Arimoclomol

2024’s novel drug approvals offered a wide array of new cancer drugs, rare disease treatments, a potential Alzheimer’s blockbuster, and about 10 fewer approvals than 2023. While 2022 and 2023 saw major approvals of new cell and gene therapies, 2024 did not stick as cleanly to a theme — instead featuring a grab-bag of long-awaited approvals. That included Eli Lilly’s amyloid-targeted Alzheimer’s drug, the first two treatments for the rare Niemann-Pick disease, and the first-ever mesenchymal stromal cell therapy. Several approvals in 2024 also deployed new ways to treat familiar diseases. For instance, Pfizer’s new hemophilia A or B drug Hympavzi is the first to target a particular protein in the blood-clotting process. Bristol Myers Squibb’s Cobenfy, approved for schizophrenia, is the first antipsychotic that targets cholinergic receptors and not dopamine receptors. What to watch for in 2025: more cancer drug approvals and major label expansions. And answers to questions like: Will the incoming Trump administration push to overturn some drug and vaccine approvals or EUAs? Or attempt to make the approval process more difficult? — Zachary Brennan Active ingredient: berdazimer sodium Indication: Molluscum contagiosum in adults and pediatric patients 1 year of age and older Snapshot : Ligand Pharmaceuticals scored FDA approval in January for berdazimer sodium, marketed as Zelsuvmi, to treat the viral skin infection molluscum contagiosum. The topical gel was approved for at-home treatment in both adult and pediatric patients ages 1 one year and older and is the first novel drug for these types of infections. The approval came after Ligand acquired the assets of Novan Pharmaceuticals, which had initially overseen the drug’s development and filed for bankruptcy in July 2023. — Lia DeGroot Active ingredient: lifileucel Indication: Adult patients with unresectable or metastatic melanoma who have been previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor. Snapshot : An idea more than 40 years in the making finally went commercial in February with the approval of the first treatment based on tumor-infiltrating lymphocytes. Iovance’s bespoke therapy, Amtagvi, is made by dissecting out immune cells from a patient’s tumor, bolstering their numbers in a lab, and reinfusing the cells into a patient to help keep cancer at bay. The company is testing the approach in other cancers and as a frontline therapy for melanoma. The Biosecure Act threatened Iovance’s relations with the Chinese contract manufacturer WuXi, which helped make Amtagvi, but sales of the therapy were still off to a strong start with $54.9 million in total revenue by the end of the third quarter. — Ryan Cross Active ingredient: cefepime and enmetazobactam Indication: Complicated urinary tract infections in patients 18 years and older Snapshot : Exblifep, a combination of cefepime and enmetazobactam delivered intravenously, was approved in February for adults with complicated urinary tract infections, including pyelonephritis, or kidney infection. Enmetazobactam was discovered by Orchid Pharma, which licensed the ex-India rights to Allecra in 2013. — Nicole DeFeudis Active ingredient: letibotulinumtoxinA-wlbg Indication: To treat moderate-to-severe frown lines in adults Snapshot : Hugel Aesthetics’ Botox competitor Letybo was approved in March for the treatment of moderate to severe frown lines. Letybo was initially rejected in 2022 over manufacturing issues. The FDA turned it down again the following year, and Hugel refiled it in September 2023 after “completing a review of the facility management and supplementary work.” Despite a growing number of Botox rivals on the market, AbbVie’s chief commercial officer Jeffrey Stewart said at the 2024 JP Morgan Healthcare Conference that the company has been “incredibly efficient” at maintaining Botox’s lead. — Nicole DeFeudis Active ingredient: resmetirom Indication: Noncirrhotic non-alcoholic steatohepatitis (NASH) with moderate-to-advanced liver scarring in adults Snapshot : Madrigal Pharmaceuticals’ resmetirom won accelerated approval in March as the first treatment for metabolic dysfunction-associated steatohepatitis (MASH), the debilitating liver disease also called NASH. The approval is likely to be seen as a crucial milestone in MASH, where the standard of care was previously diet and exercise. Rezdiffra, as the drug is marketed, is indicated for patients with stage 2 or 3 fibrosis who have not progressed to cirrhosis. — Nicole DeFeudis Active ingredient: tislelizumab Indication: Unresectable or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic chemotherapy that did not include a PD-(L)1 inhibitor Snapshot : BeiGene had licensed US rights to its PD-1 inhibitor twice during the development journey, once to Celgene and the other time to Novartis. By the time the biotech clinched FDA approval for Tevimbra, it became the sole owner again. While Novartis and BeiGene had first filed for approval more than two years prior, the FDA was unable to inspect BeiGene’s manufacturing site in China amid the pandemic, leading to a lengthy review delay. As with the many checkpoint drugs already on the market, BeiGene hopes esophageal cancer will be the first of many indications Tevimbra will eventually be approved to treat, both on its own and in combination with other medicines. The drug was first approved in China in 2019. — Amber Tong Active ingredient: atidarsagene autotemcel Indication: Children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy Snapshot : With a $4.25 million price tag, Lenmeldy is on the list of the world’s most expensive medicines. The gene therapy is for an ultra-rare and fatal disease called early-onset metachromatic leukodystrophy, which affects fewer than 40 children a year in the US. Lenmeldy was already approved under a different brand name in the EU and UK and was shown in clinical trials to keep the majority of children who received it alive and with normal cognitive development. Japanese pharmaceutical company Kyowa Kirin completed its purchase of the company that developed the therapy, Orchard Therapeutics, last January after Orchard had been struggling to build a sustainable business around commercializing these complex treatments for rare diseases with small numbers of patients. — Lei Lei Wu Active ingredient: aprocitentan Indication: Hypertension in combination with other antihypertensive drugs, to lower blood pressure in adult patients who are not adequately controlled on other drugs Snapshot : The FDA approved Idorsia’s Tryvio for patients who aren’t adequately treated by current blood pressure medications. Johnson & Johnson backed away from its partnership on the drug in 2022, and while the pharma will still receive royalties, Idorsia needs a new partner to sustain its operations through this year. Idorsia said in November that it entered exclusive negotiations with an undisclosed third party for global rights to the drug, netting $35 million, and that it could lay off approximately 270 staffers. The drug carries a black box warning for embryo-fetal toxicity and is only available via a restricted safety program. The FDA only approved the lower dose that Idorsia studied in its key clinical trial. — Lei Lei Wu Active ingredient: givinostat Indication: Duchenne muscular dystrophy in patients 6 years of age and older. Snapshot : The FDA’s approval of Duvyzat marked the first commercial nonsteroidal drug for all genetic variants of Duchenne muscular dystrophy. The treatment, an oral histone deacetylase inhibitor developed by Italfarmaco, was approved for Duchenne patients aged 6 and older. Unlike genetic medicines for Duchenne, such as exon-skipping drugs or gene therapy, Duvyzat aims to counteract the downstream effects of missing dystrophin, a crucial muscle protein, rather than trying to restore it. In clinical trials, Duvyzat led to improved performance on a stair climb test that’s used to measure motor function in patients with muscle diseases. — Lei Lei Wu Active ingredient: sotatercept Indication: Pulmonary arterial hypertension in adults Snapshot : Merck’s $11.5 billion Acceleron deal appeared to pay off with the much-anticipated approval of its pulmonary arterial hypertension drug Winrevair. The rare disease medicine, which had a “successful resurrection” around mid-stage data in 2020, is expected to become a quick blockbuster treatment as the New Jersey pharma looks to fill the eventual patent gap with Keytruda. The under-the-skin drug, when combined with background PAH therapy, helped patients walk farther than patients on placebo and background treatment in a Phase 3 trial. “The increase in six-minute walk distance capability was gargantuan,” Eliav Barr, chief medical officer at Merck Research Laboratories, said in an interview with Endpoints News . — Kyle LaHucik Active ingredient: vadadustat Indication: Anemia from chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months Snapshot : After securing approval in more than 35 other countries, Akebia’s anemia drug finally got the FDA’s blessing in March, almost two years to the day after it was initially rejected by the agency. The once-daily Vafseo tablets were greenlit for adults who have been on dialysis for at least three months and indicated for anemia due to chronic kidney disease. The drug belongs to a class of medicines known as hypoxia-inducible factor prolyl hydroxylase inhibitors. They’ve had a precarious history. In 2021, FibroGen’s drug was also rejected by the FDA. GSK, meanwhile, won US approval in February 2023, but wound up ditching plans to seek European approval. Akebia doesn’t plan on making the drug available in the US until this month. — Kyle LaHucik Active ingredient: danicopan Indication: Add-on therapy for extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH) Snapshot : The oral factor D inhibitor Voydeya from AstraZeneca scored approval in the US in April 2024 as an add-on therapy to Soliris and Ultomiris for the treatment of a rare blood disorder called extravascular hemolysis. Voydeya is also being developed as a monotherapy for an advanced form of age-related macular degeneration. — Katherine Lewin Active ingredient: ceftobiprole Indication: Adults with Staphylococcus aureus bloodstream infections; adults with acute bacterial skin and skin structure infections; and adult and pediatric patients 3 months to less than 18 years old with community-acquired bacterial pneumonia Snapshot : The FDA approved Zevtera for staph infections, acute bacterial skin and skin structure infections, and community-acquired bacterial pneumonia. The approval came later than planned — Basilea initially said that it would submit the drug to the FDA in early 2023, but pushed back those plans after manufacturing issues. — Katherine Lewin Active ingredient: nogapendekin alfa inbakicept-pmln Indication: Adult patients with BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumors Snapshot : Anktiva was approved in combination with Bacillus Calmette-Guérin (BCG) to treat patients with a subtype of BCG-unresponsive non-muscle invasive bladder cancer. ImmunityBio was hit with an FDA rejection in May 2023, citing manufacturing issues with a third party. After the eventual approval, ImmunityBio received $100 million as part of a royalty deal and private placement with Oberland Capital. — Katherine Lewin Active ingredient: tovorafenib Indication: Children 6 months of age and older with low-grade glioma that has stopped responding to or returned after previous treatment Snapshot : Day One Biopharmaceuticals’ goal is to develop new drugs for children first — reversing the industry’s age-old practice of first winning approval of medicines in adults and then (and only on occasion) in children. The company’s first approved drug, Ojemda, is a targeted treatment called a BRAF inhibitor. It was approved for children 6 months of age and up with a form of slow-growing brain cancer called low-grade glioma. “We now have shown that you can develop drugs intentionally for children at the speed of biotech,” Day One’s then-R&D chief Sam Blackman told Endpoints around Ojemda’s approval. — Lei Lei Wu Active ingredient: pivmecillinam Indication: Uncomplicated urinary tract infections caused by susceptible isolates of Escherichia coli , Proteus mirabilis and Staphylococcus saprophyticus , in female patients 18 years and older Snapshot : In April, Utility Therapeutics’ Pivya became the first new treatment for uncomplicated urinary tract infections in more than two decades. The oral antibiotic induced a composite response in 62% of patients versus just 10% for placebo in one of Utility’s three registrational trials. The UK-based drugmaker bought the US rights to pivmecillinam — the active ingredient in Pivya — from Leo Pharma as part of a 2018 deal with undisclosed financial terms. — Ayisha Sharma Active ingredient: fidanacogene elaparvovec-dzkt Indication: Moderate-to-severe hemophilia B in adults Snapshot : Pfizer became the second company to nab FDA approval for a one-time gene therapy to treat hemophilia B. And just like the US market’s first entrant, CSL, Pfizer priced the treatment at an eye-popping $3.5 million. Pfizer’s Beqvez was found to eliminate bleeding in 60% of patients in an open-label Phase 3 study, compared to 29% of patients receiving prophylaxis with a factor IX inhibitor. While it won approval for Beqvez, Pfizer has taken a step back from the gene therapy field, selling early assets in 2023 and dropping its Duchenne gene therapy after a failed trial. — Lei Lei Wu Active ingredient: mavorixafor Indication: WHIM syndrome in patients 12 and older Snapshot : Xolremdi was approved for an ultra-rare immune disorder, WHIM, named for its symptoms: warts, hypogammaglobulinemia, infections, and myelokathexis. X4 Pharmaceuticals’ drug is a CXCR4 inhibitor that is meant to address the underlying cause of the disease and improve patients’ ability to fight infections. X4 is also exploring Xolremdi’s use in treating chronic neutropenia. — Lei Lei Wu Active ingredient: tarlatamab-dlle Indication: Extensive-stage small cell lung cancer with disease progression on or after platinum-based chemotherapy. Snapshot : Imdelltra’s approval marked a major milestone for bispecific T cell engagers, a class of antibody-based drugs that bring T cells to tumors in hopes that they will attack the cancer. Following Immunocore’s Kimmtrak, it became the second T cell engager approved to treat solid tumors. Imdelltra was approved for extensive-stage small cell lung cancer that progressed despite chemotherapy, and Wall Street analysts project that the therapy has blockbuster potential if it can move into earlier lines of treatment. — Lei Lei Wu Active ingredient: RSV vaccine Indication: Prevention of lower respiratory tract disease caused by respiratory syncytial virus (RSV) in individuals 60 and older Snapshot : Moderna expects its RSV vaccine, its first non-Covid approval, to play a significant role in the company’s return-to-growth strategy by 2026 and reverse the $4.7 billion loss it recorded in 2023. The first full RSV vaccine season (winter 2023-24) was highly successful for Moderna’s competitors, GSK and Pfizer, and analysts anticipate the market will grow to $10 billion over the next few years. — Max Gelman Active ingredient: imetelstat Indication: Adults with low- to intermediate- risk myelodysplastic syndromes with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents Snapshot : Geron’s imetelstat cleared an adcomm and then secured an FDA nod in June for certain anemic myelodysplastic syndrome patients who are dependent on blood transfusions. Branded as Rytelo, the telomerase inhibitor carries a $2,471 price tag for a 47 mg vial and costs $9,884 for a 188 mg vial. The approval came after a panel of experts voted 12 to 2 in March that the drug’s benefits outweigh its risks. — Ayisha Sharma Active ingredient: elafibranor Indication: Primary biliary cholangitis in combination with ursodeoxycholic acid in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA Snapshot : Ipsen’s $136 million bet on Genfit’s elafibranor paid off in June when the drug clinched US approval for primary biliary cholangitis, a rare disease that can lead to liver failure. The drug, sold as Iqirvo, is a first-in-class dual agonist of the alpha and delta isoforms of PPAR and could slow disease progression, according to Ipsen. PBC affects around 100,000 people in the US. — Ayisha Sharma Active ingredient: immune globulin intravenous, human-dira Indication: Primary humoral immunodeficiency (PI) in patients 2 or older Snapshot : Yimmugo was approved in June for patients 2 years and older with primary humoral immunodeficiency, a condition that affects the immune system and can leave patients at an increased risk of infections. Italy-based Kedrion Biopharma announced in July that it struck an agreement for the full commercialization and distribution of Yimmugo in the US. — Nicole DeFeudis Active ingredient: pneumococcal 21-valent conjugate vaccine Indication: For prevention of invasive pneumococcal disease and pneumococcal pneumonia. Snapshot : Merck’s 21-valent pneumococcal vaccine Capvaxive was specifically designed for adults. The strains covered by the vaccine were responsible for 84% of invasive pneumococcal disease in patients 50 years and older between 2018 and 2021, according to Merck. That includes 10 strains in common with Pfizer’s rival 20-valent shot Prevnar 20, plus 11 unique strains. — Nicole DeFeudis Active ingredient: sofpironium Indication: Primary axillary hyperhidrosis in adult and pediatric patients 9 years of age and older Snapshot : Botanix Pharmaceuticals unlocked a potential market of 10 million people in the US with the June approval of sofpironium for primary axillary hyperhidrosis, or excessive underarm sweat. Sold as a gel with the brand name Sofdra, Botanix launched an early patient experience program in the third quarter, followed by a broader launch in the fourth quarter, which is when it also expected its first revenues from the product. — Ayisha Sharma Active ingredient: crovalimab-akkz Indication: Adult and pediatric patients 13 years and older with paroxysmal nocturnal hemoglobinuria and body weight of at least 40 kg Snapshot : The FDA approval for Genentech’s C5 inhibitor crovalimab for people with paroxysmal nocturnal hemoglobinuria in June came with a significant caveat — the drug can only be given under a REMS pathway due to the serious risk of meningococcal infections. Two months after its US approval, crovalimab, sold as PiaSky, secured an EU green light for the rare disease. When left untreated, PNH causes intravascular hemolysis, thromboembolic events and cytopenia. — Ayisha Sharma Active ingredient: ensifentrine Indication: Maintenance treatment of chronic obstructive pulmonary disease in adult patients Snapshot : Verona Pharma launched its COPD drug Ohtuvayre in 2024, taking a step into a titan-filled arena. Known generically as ensifentrine, Ohtuvayre is a maintenance treatment for COPD, taken via nebulizer twice a day, that can be added to standard COPD therapies. Verona aims to carve out a niche for Ohtuvayre as an add-on treatment rather than competing directly with major pharma companies in COPD like GSK and AstraZeneca. — Lei Lei Wu Active ingredient: donanemab Indication: Alzheimer’s disease Snapshot : Eli Lilly has invested more than $8 billion over more than three decades in its search for an Alzheimer’s drug. It finally found success in July when the FDA approved Kisunla . Kisunla is the second amyloid-targeting antibody to get full FDA approval after Eisai and Biogen won approval for Leqembi last year. Experts once worried about Alzheimer’s drugs bankrupting Medicare, but sales of the drugs are off to a slower-than-expected start , partly due to the risk of potentially fatal brain bleeding and swelling — which occurred in about 1 in 300 people in Lilly’s study. Lilly hopes the drug will be safer and more effective when taken earlier, before symptoms of dementia begin. A large study testing that hypothesis is underway and is expected to wrap up in 2027. — Ryan Cross Active ingredient: deuruxolitinib Indication: Adults with severe alopecia areata Snapshot : Sun Pharma’s $576 million acquisition of Concert Pharmaceuticals in 2023 paid off with the approval of the JAK inhibitor Leqselvi in severe alopecia areata. Sun’s drug could compete against some big players, including Eli Lilly’s JAK inhibitor Olumiant and Pfizer’s Litfulo. However, the drug’s launch was delayed after Incyte, which makes a similar drug, secured an injunction against its sale in November. Sun said it planned to appeal. — Katherine Lewin Active ingredient: benzgalantamine Indication: Mild to moderate dementia of the Alzheimer’s type in adults Snapshot : Zunveyl is a new twist on an old pill that’s commonly used to help reduce some symptoms of Alzheimer’s disease, but potentially with fewer gastrointestinal side effects. The compound is a prodrug of galantamine, an acetylcholinesterase inhibitor approved in 2001 that blocks breakdown of a key neurochemical that brain cells use to communicate with each other. Alpha Cognition, a tiny Canadian company that won approval for Zunveyl in July based on bioavailability studies, said it will begin selling Zunveyl in early 2025. — Ryan Cross Active ingredient: afamitresgene autoleucel Indication: Unresectable or metastatic synovial sarcoma in adults have received prior chemotherapy, are HLA antigen(s) A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive, and whose tumor expresses the MAGE-A4 antigen as determined by FDA authorized companion diagnostic devices. Snapshot : Tecelra became the first cell therapy based on T cell receptors and the second cell therapy for a solid tumor cancer to win approval in the US in 2024. The therapy, known as afami-cel, is meant for a subsection of synovial sarcoma patients whose cancer has spread throughout their body or can’t be removed via surgery, and who have already received chemotherapy. Patients need to be tested for expression of a protein called MAGE-A4, which is found in a range of tumors, as well as an immune marker called HLA-A*02. — Lei Lei Wu Active ingredient: vorasidenib Indication: For adult and pediatric patients 12 years and older with grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, following surgery including biopsy, sub-total resection, or gross total resection. Snapshot : Brain cancer is getting its targeted therapies, too. Servier’s Voranigo was approved for people with slow-growing gliomas that bear an IDH mutation. Patients are eligible for the drug after surgery for their brain tumor. Over 80% of grade 2 gliomas bear an IDH mutation, and the FDA said this was the first approved systemic drug for this subset of patients. In a clinical trial versus placebo, the treatment staved off cancer progression for a median 16.6 months longer, which physicians called a “striking” result. — Lei Lei Wu Active ingredient: denileukin diftitox-cxdl Indication: Adult patients with relapsed or refractory stage I-III cutaneous T cell lymphoma after at least one prior systemic therapy Snapshot : Following a delay from a CRL over manufacturing issues in 2023, Lymphir gained FDA approval in August. In order to support the launch of the treatment for a rare cancer, the company’s cancer subsidiary, Citius Oncology, combined with TenX Keane Acquisition. — Katherine Lewin Active ingredient: palopegteriparatide Indication: Hypoparathyroidism in adults Snapshot : Ascendis rebounded from an earlier FDA rejection and subsequent delay of its new application to snag FDA approval of hypoparathyroidism drug Yorvipath. It was already approved in Austria and Germany, generating €1.5 million ($1.6 million) in the first quarter. Yorvipath’s US entry replaces a void left by Takeda’s Natpara, some two years after it was pulled off the market. Ascendis said at the time of approval that it was completing manufacturing of the product and expecting the first batch of supply to be available in the first quarter of 2025. — Max Bayer Active ingredient: nemolizumab Indication: Prurigo nodularis in adults Snapshot : In August, Galderma secured first-in-class status with the US approval of its IL-31 antibody nemolizumab for prurigo nodularis. The green light is the first step in unlocking Nemluvio’s blockbuster potential, much of which lies in eczema, according to Galderma. The Swiss drugmaker earned a label expansion for moderate-to-severe atopic dermatitis in December, with Jefferies analysts projecting $2.1 billion in global peak sales for the drug. — Ayisha Sharma Active ingredient: seladelpar Indication: Primary biliary cholangitis in combination with ursodeoxycholic acid, in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA Snapshot : After acquiring seladelpar in the $4.3 billion purchase of CymaBay Therapeutics, Gilead snagged FDA accelerated approval to treat PBC, boosting its liver portfolio. The drug joins three others on the market, though its ability to treat itch symptoms gives Gilead hope that it can edge out competitors. That potential benefit has also contributed to Livdelzi’s price, which is slightly higher than the other two approved therapies. — Max Bayer Active ingredient: axatilimab-csfr Indication: Chronic graft-versus-host disease after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg Snapshot : Thanks to the help of Syndax Pharmaceuticals, Incyte was able to get another cGVHD treatment approval in August. It already markets Jakafi. The newly approved CSF-1 receptor-blocking antibody will also be studied alongside Jakafi and in combination with steroids. Incyte has said it plans to target the approximately 6,500 people who have failed two prior drugs. It also hopes to get the medicine greenlit for idiopathic pulmonary fibrosis. — Kyle LaHucik Active ingredient: lazertinib Indication: Locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor exon 19 deletions or exon 21 L858R substitution mutations, as detected by an FDA-approved test Snapshot : Lazcluze was approved in August in combination with Johnson & Johnson’s Rybrevant for certain non-small cell lung cancer patients with two common EGFR mutations. The approval puts the combination therapy in direct competition with AstraZeneca and its blockbuster Tagrisso, which has been approved in the same lung cancer population since 2018. Tagrisso also recently won a label expansion as part of a chemotherapy combination regimen in that indication. — Nicole DeFeudis Active ingredient: lebrikizumab Indication: Moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable, in adult and pediatric patients 12 years and older who weigh at least 40 kg Snapshot : Following an FDA rejection over manufacturing issues in 2023 (and no concerns with safety or labeling), Lilly scored approval for Ebglyss in September for moderate-to-severe eczema. Between two studies, 38% of patients on Lilly’s drug achieved clear or almost-clear skin at 16 weeks. — Katherine Lewin Active ingredient: arimoclomol Indication: Neurological manifestations of Niemann-Pick disease type C in adult and pediatric patients 2 and older, in combination with miglustat Snapshot : Zevra Therapeutics won approval for Miplyffa, known generically as arimoclomol, for people with the rare genetic disorder Niemann-Pick disease type C who are aged 2 years and older. Miplyffa’s approval came after the FDA previously rejected the drug in 2021, but Zevra returned to ask for approval with a reanalysis of clinical trial data that showed improvements in swallowing, which can be affected by the disease. FDA hosted an advisory committee ahead of its decision that voted in favor of the drug for the disease, a lysosomal storage disorder in which the body can’t properly recycle certain fatty substances. — Lei Lei Wu Active ingredient: levacetylleucine Indication: Neurological manifestations of Niemann-Pick disease type C in adults and pediatric patients weighing ≥15 kg Snapshot : In September, IntraBio’s levacetylleucine became the second drug to clinch FDA approval for Niemann-Pick disease type C in the span of just one week. Branded Aqneursa, the oral drug can be taken up to three times daily with weight-dependent dosing. — Ayisha Sharma Active ingredient: xanomeline and trospium chloride Indication: Schizophrenia in adults Snapshot : Cobenfy’s approval gives Bristol Myers a significant foothold in the neuroscience and psychiatry fields, as it brings to market the first new schizophrenia drug in decades. It also validates the company’s $14 billion acquisition of Karuna, with execs predicting peak sales in the multibillion-dollar range. Researchers hope to broaden the use of Cobenfy in at least half a dozen other indications. — Max Gelman Active ingredient: inavolisib Indication: For adults with endocrine-resistant, PIK3CA-mutated, hormone receptor-positive, human epidermal growth factor receptor 2-negative, locally advanced or metastatic breast cancer, as detected by an FDA-approved test, following recurrence on or after completing adjuvant endocrine therapy Snapshot : Roche’s Itovebi, which the company says is the first targeted therapy approved for people with HR-positive disease, got the green light from the FDA in October for patients with PIK3CA-mutated HR-positive HER2-negative breast cancer in combo with chemotherapy and Pfizer’s Ibrance. Patients on the drug saw progression-free survival of 15 months and a 58% overall response rate. — Katherine Lewin Active ingredient: marstacimab-hncq Indication: For routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years and older with: hemophilia A without factor VIII inhibitors, or hemophilia B without factor IX inhibitors Snapshot : Pfizer had a few successes in hemophilia in 2024, first with a gene therapy approval in April and then the nod for Hympavzi. The drug reduces a naturally occurring anticoagulation protein, unlike other treatments, which replace a clotting factor. But the results over routine prophylaxis were “modest,” TD Cowen analysts have said. Pfizer will likely have to rely on its packaging as a pre-filled, auto-injector pen to get traction in the market, the analysts said. — Kyle LaHucik Active ingredient: zolbetuximab Indication: In combination with fluoropyrimidine- and platinum-containing chemotherapy for the first-line treatment of adults with locally advanced unresectable or metastatic human epidermal growth factor receptor 2-negative gastric or gastroesophageal junction adenocarcinoma whose tumors are Claudin18.2-positive, as determined by an FDA-approved test Snapshot : Vyloy, which targets a protein called Claudin18.2, scored an approval in combination with chemotherapy for the first-line treatment of locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma. The drug was approved in Europe and Japan as well, making it the first in its class to get worldwide approval. — Katherine Lewin Active ingredient: sulopenem etzadroxil and probenecid Indication: Uncomplicated urinary tract infections caused by the designated microorganisms Escherichia coli , Klebsiella pneumoniae , or Proteus mirabilis in adult women who have limited or no alternative oral antibacterial treatment options Snapshot : Iterum’s oral sulopenem was approved in October for certain patients with uncomplicated urinary tract infections (uUTIs). The drug, to be marketed as Orlynvah, is indicated specifically for patients who have limited or no alternative oral options. While the company had applied for broad use in adults with uUTIs, Iterum CEO Corey Fishman said the FDA’s label restrictions are “very consistent with how we’ve been thinking the whole time.” The agency had previously raised concerns that “inappropriate and/or extensive use” of an oral penem could result in cross-resistance with carbapenems, a class of antibiotics used for severe infections. — Nicole DeFeudis Active ingredient: obecabtagene autoleucel Indication: Adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia Snapshot : Autolus is challenging one of Gilead’s CAR-T cell therapies, Tecartus, with its recently approved treatment Aucatzyl in adult B-ALL. Aucatzyl will be listed at $525,000, making it pricier than Tecartus, but Autolus is hoping that its therapy’s safety profile will win over payers and providers. — Lei Lei Wu Active ingredient: eladocagene exuparvovec-tneq Indication: Adult and pediatric patients with aromatic 13 L-amino acid decarboxylase deficiency Snapshot : Known as Upstaza in Europe and Israel, PTC Therapeutics’ rare disease gene therapy finally won approval in the US in November under the brand name Kebilidi. The gene therapy is for AADC deficiency, an ultra-rare disease that impacts the nerve cells and brain. In the US, the therapy’s approval included children and adults across “the full spectrum of disease severity,” whereas the treatment’s previous approvals outside the country included patients with severe AADC deficiency. — Lei Lei Wu Active ingredient: revumenib Indication: Relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene translocation in adult and pediatric patients 1 year and older Snapshot : Revuforj, also known as revumenib, is the first drug approved in a new class of medicines called menin inhibitors. It’s used for an aggressive form of leukemia driven by rearrangement of the KMT2A gene. However, it carries a boxed warning for differentiation syndrome, a potentially life-threatening reaction to some blood cancer treatments. Syndax plans to seek approval in 2025 for leukemia patients with a different and more common genetic alteration called NPM1. — Nicole DeFeudis Active ingredient: zanidatamab Indication: Adults with previously treated, unresectable or metastatic HER2-positive biliary tract cancer (BTC), as detected by an FDA-approved test Snapshot : Jazz Pharmaceuticals’ HER2-targeted bispecific antibody zanidatamab will be the first of its class to hit the market for biliary tract cancer. The drug, to be marketed as Ziihera, won accelerated approval in November for previously treated patients who scored high on an immunohistochemistry test measuring HER2 expression. A Phase 3 confirmatory trial is underway in the first-line setting, which executives say could support both a full approval in the second-line setting and a supplemental application for frontline patients. — Nicole DeFeudis Active ingredient: acoramidis Indication: Cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis in adults to reduce cardiovascular death and cardiovascular-related hospitalization Snapshot : Attruby, approved in November, is expected to compete with Pfizer’s Vyndaqel franchise and Alnylam’s Amvuttra, should the FDA greenlight the latter ahead of its March 23 decision date. BridgeBio and Alnylam also traded blows as the drugs moved through the clinic (Vyndaqel was approved in 2019). Notably, Attruby’s label said the drug reduces the likelihood of both hospitalization and death, putting it on the same ground as Vyndaqel. — Max Gelman Active ingredient: zenocutuzumab-zbco Indication: Pancreatic adenocarcinoma or NSCLC that harbors NRG1 gene fusions and are advanced unresectable or metastatic Snapshot : The new cancer drug marked the first commercial product for Merus. But it will be Partner Therapeutics that actually commercializes the product in the US, after paying for rights in 2024. — Max Bayer Active ingredient: cosibelimab-ipdl Indication: Adults with locally advanced or metastatic cutaneous squamous cell carcinoma who cannot receive curative surgery or radiation Snapshot : A new PD-L1 drug in 2024? Apparently so. Checkpoint Therapeutics won approval late in the year for its checkpoint inhibitor cosibelimab, which will be branded as Unloxcyt. The drug won approval for a form of advanced skin cancer, where Merck’s Keytruda and Regeneron’s Libtayo are already approved. Checkpoint was part of an earlier wave of companies that wanted to disrupt how the blockbuster class of cancer drugs are priced by selling their treatments at a discount compared to currently approved options, but those aspirations have failed to become reality. Checkpoint now says it believes its drug can be priced “at or near parity with other approved anti-PD-(L)1 therapies,” and the company is seeking a partner or business deal before launching the drug commercially. — Lei Lei Wu Active ingredient: crinecerfont Indication: To be used together with glucocorticoids to control androgen levels in adults and pediatric patients 4 years and older with classic congenital adrenal hyperplasia Snapshot : Neurocrine’s second approved product marked the first new drug for congenital adrenal hyperplasia in 70 years. It also brings some of the work of the company’s late founder, Wylie Vale, to fruition: Vale, who died in 2012, identified the compound that this drug works to block. Analysts at William Blair believe the drug could reach nearly $1.5 billion in peak sales by 2030. — Max Bayer Active ingredient: ensartinib Indication: Adult patients with anaplastic lymphoma kinase-positive locally advanced or metastatic non-small cell lung cancer who have not previously received an ALK inhibitor Snapshot : Xcovery won its first-ever FDA approval for a first-line treatment of adults with anaplastic lymphoma kinase (ALK)-positive locally advanced or metastatic non-small cell lung cancer. The approval was based on a trial of 290 patients who had not previously received an ALK-targeted therapy, and FDA said Ensacove demonstrated a statistically significant progression-free survival improvement compared to Pfizer’s Xalkori. Xcovery is also testing Ensacove in other cancers. — Zachary Brennan Active ingredient: remestemcel-L-rknd Indication: Steroid-refractory acute graft-versus-host disease in pediatric patients 2 months and older Snapshot : Ryoncil took a winding path to approval, including two rejections and concerns from regulators about the design of its single-arm trial. An approval came more than four years after regulators accepted Mesoblast’s first application. But CEO Silviu Itescu believes it was worth the wait. Ryoncil is now the first mesenchymal stromal cell therapy to be approved by the FDA. It will be available for pediatric patients 2 months and older with acute graft-versus-host disease, a complication of allogeneic transplant in which donor cells attack the recipient. — Nicole DeFeudis Active ingredient: olezarsen Indication: As an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome Snapshot : Ionis’ antisense oligonucleotide olezarsen is the first approved treatment for familial chylomicronemia syndrome, a rare metabolic disorder that prevents the body from breaking down fats called triglycerides. The approval marks an important transition for Ionis, as Tryngolza will be the company’s first independent launch following years of partnerships. While FCS affects few people in the US, CEO Brett Monia hopes that a potential label expansion to a broader condition called severe hypertriglyceridemia could make Tryngolza a blockbuster. — Nicole DeFeudis Active ingredient: concizumab-mtci Indication: For routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A or B with inhibitors Snapshot : The anti-tissue factor pathway inhibitor Alhemo finally came good for Novo Nordisk at the tail end of 2024 following its rejection by the FDA in May 2023. It is the first daily subcutaneous treatment for hemophilia A or B patients with inhibitors — antibodies that neutralize clotting factors — to be delivered via a pen injector. It will compete with Pfizer’s similar weekly shot Hympavzi, but could reach more patients since Hympavzi is only approved for hemophilia A and B patients without inhibitors. — Elizabeth Cairns Active ingredient: vanzacaftor, tezacaftor, and deutivacaftor Indication: Cystic fibrosis in patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene Snapshot : Alyftrek is Vertex’s “vanza triple” combination treatment for cystic fibrosis, a modulator of the CFTR gene. It’s the fifth such therapy Vertex has pushed across the finish line, but it comes with a boxed warning for potential drug-induced liver injury and failure. The treatment succeeded in its Phase 3 trials earlier this year, and Vertex used a priority review voucher to reduce the FDA’s review time to six months. — Max Gelman