Plozasiran

The FDA in 2025 is set to approve its lowest number of new drug and biologic applications since 2022, even as more than a dozen companies await the regulator’s decisions on their new molecular entities before the end of the year. The number of drug approvals isn’t always indicative of the quality of innovation or whether major strides are being made in hard-to-treat diseases. But it does provide an indication of R&D performance year-over-year. It’s also unclear whether losing thousands of FDA staff played a role in slowing down the approval process this year. The FDA has approved 42 new drugs and biologics so far this year, with about 16 decisions to go before 2026, according to an Endpoints News tally . In comparison, the regulator approved 69 new drugs and biologics in 2024, and 80 in 2023. But it was 2022 that had the fewest number of new approvals since 2020, with 51 new drugs and biologics approved. In 2020, the FDA approved 61 new drugs and biologics, and 63 in 2021. An FDA spokesperson told Endpoints News that “drug reviews and approvals are not affected by the Democrat-led shutdown and this work continues.” This year’s approval numbers likely won’t hit historic highs if applications continue to be delayed at the same rate as they have been recently . Back in July, FDA Commissioner Marty Makary told CNBC that the agency “could hit a record number of approvals” in 2025. The FDA spokesperson did not comment on whether that prediction still stands. Hello, Craig Smith! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Craig Smith! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Craig Smith! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Craig Smith! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Craig Smith! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Craig Smith! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Craig Smith! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Craig Smith. You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. At least 18 companies are still waiting for the FDA to decide whether to approve their new drug or biologic applications this year:

iStock, Grandbrothers From more than 30 target action dates in the last three months of the year, BioSpace has narrowed the list to six regulatory decisions that could have far-reaching implications for biopharma and patients. As 2025 enters its home stretch, the FDA is also nearing the end of its calendar. But that doesn’t mean that the industry is in for a quiet year-end—quite the opposite, in fact. In the last three months of 2025, the FDA is set to decide on at least 30 regulatory applications, many of which could help shift the treatment landscape for hard-to-treat diseases or push the boundaries for novel modalities. One upcoming verdict, for instance, could mark another industry first for CAR T therapies, while another could mean the comeback of a previously-pulled cancer drug. Here, BioSpace rounds up six of these most highly anticipated decisions in Q4, looking at the data backing their bids and what their approvals could mean for the industry. After Delay, Regeneron Awaits Two Eylea HD Verdicts Regeneron is looking to boost the pro high-dose Eylea with a move into macular edema after retinal vein occlusion (RVO) and a monthly dosing regimen across all its approved indications. The FDA is currently reviewing these applications and is expected to release its verdict in the fourth quarter. No specific decision date has yet been indicated. The regulator had an initial target action date of Aug. 19, but extended the review period due to issues at a third-party manufacturing site, Regeneron announced earlier that month. The production plant in contention was previously owned by the CDMO giant Catalent, which was acquired by Novo Nordisk’s parent company for $16.5 billion in February 2024 . An FDA report made public in August revealed uncontrolled and unaddressed contaminations at this site, including with bacteria and cat hair. Regeneron has nevertheless reassured investors that aside from these third-party problems, the FDA has not flagged any problems with the submission. “Based on our discussions, we believe that there’s nothing significant left to be done,” CEO Leonard Schleifer said during the company’s Q2 earnings call . “We are expecting, once the resolution of the filling issues has occurred, to receive favorable action, we hope, from the FDA.” To support its application for high-dose Eylea, Regeneron filed data from the Phase III QUASAR study, which demonstrated that the high-dose formulation was non-inferior to the currently approved standard-dose version of Eylea when used to treat patients with macular edema after RVO, according to a readout in December 2024. Adverse events were comparable between the two formulations. High-dose Eylea is currently approved for wet age-related macular degeneration, diabetic macular edema and diabetic retinopathy. GSK’s Blenrep Comeback Bid Nears Moment of Truth By Oct. 23, the FDA is expected to release its decision on GSK’s proposal to bring Blenrep back to the market, this time as a second-line treatment for relapsed/refractory multiple myeloma. Blenrep, an antibody-drug conjugate targeting the CD38 protein, was originally approved in August 2020 under the FDA’s accelerated pathway. But soon after failing its confirmatory Phase III study in November 2022, GSK withdrew the product from the market. Then, last year, GSK started setting the stage for Blenrep’s comeback with data from the Phase III DREAMM-7 trial. A readout in February 2024 demonstrated a significant 59% drop in the risk of death or disease progression in patients treated with Blenrep plus bortezomib and dexamethasone, as compared with a similar regimen using Johnson & Johnson’s Darzalex. Then, in June that same year, the pharma reported findings from another late-stage study, DREAMM-8, touting significantly better progression-free survival for Blenrep versus Takeda’s Velcade. GSK is using these two studies to support its regulatory application for Blenrep, but in July this year, the FDA’s internal reviewers flagged “high rates of ocular toxicity and dose modifications” in both trials. The DREAMM studies also had “limited dose exploration” which in turn highlight the unaddressed need for a “careful evaluation” of Blenrep’s risks in its proposed indications, according to the reviewers. An external advisory committee in July agreed with this assessment and voted 7–1 against Blenrep , finding an unfavorable benefit-risk pro the drug in its proposed indication. “The efficacy data were strong but the toxicity data were also very strong,” panelist Neil Vasan said at the time. Shortly after the vote, the FDA extended Blenrep’s review by three months. BMS’ Breyanzi Could Become First CAR T Therapy for Marginal Zone Lymphoma Bristol Myers Squibb is proposing its CAR T therapy Breyanzi for the treatment of marginal zone lymphoma (MZL), potentially blazing the trail for the therapeutic class in this indication. The FDA has a target decision date of Dec. 5. To support its application, BMS filed findings from the Phase II TRANSCEND FL study, a single-arm, open-label trial that enrolled more than 270 patients with relapsed or refractory follicular lymphoma or MZL. Data presented at the 2025 International Conference on Malignant Lymphoma in June demonstrated a 95.5% overall response rate in patients with MZL, including 62.1% who saw a complete response. The safety pro TRANSCEND FL was consistent with what had previously been established for Breyanzi. Rates of cytokine release syndrome and neurologic complications were “low,” according to BMS, with no grade 4 or 5 cases detected. If it wins the FDA’s blessing, Breyanzi would be the first CAR T therapy indicated for MZL, according to an August 2025 news release from the company. It would also mark the second time that Breyanzi notched a regulatory record: In March 2024, it became the first CAR T to win approval for chronic lymphocytic leukemia and small lymphocytic leukemia in relapsed/refractory patients. Breyanzi works by binding to the CD19 protein, in turn triggering the activation and action of CAR-T cells, as well as the release of pro-inflammatory signals to destroy the target tumor. It is also approved for large B-cell lymphoma, follicular lymphoma and mantle cell lymphoma. The drug’s label carries a boxed warning for cytokine release syndrome, neurological toxicities and secondary hematological malignancies. Arrowhead Makes Case for RNAi Therapy in Rare Cholesterol Disease After delivering four firsts for the rare disease space in August, the FDA by Nov. 18 could grant another—this one for Arrowhead Pharmaceuticals’ plozasiran , an RNA interference therapeutic for familial chylomicronemia syndrome (FCS). Afflicting 1 to 2 people per million , FCS is an ultra-rare, heritable disease that manifests as excessively high levels of triglyceride levels, typically reaching over 880 mg/dL, according to Arrowhead . In turn, patients with FCS suffer from cardiometabolic and other complications such as diabetes, hepatic steatosis, abdominal pain and even cognitive problems. There are currently no treatments that can adequately control FCS in the U.S., according to Arrowhead. Arrowhead’s answer to FCS is plozasiran , an investigational therapy that reduces the production of the ApoC3 protein, which is naturally produced in liver cells and serves as a component of triglyceride-rich lipoproteins. ApoC3 prevents the breakdown and clearance of these fatty molecules. By reducing ApoC3 production, plozasiran helps normalize lipid levels in FCS patients. Arrowhead is supporting its application with data from the Phase III PALISADE trial. A readout in November 2024 demonstrated that 25 mg of plozasiran—the proposed commercial dose—led to a roughly 80% reduction in triglyceride levels. In at least half of the treated patients, triglyceride levels stayed below 500 mg/dl, a threshold linked with worse acute pancreatitis risk. Plozasiran treatment also resulted in lower cholesterol levels. Arrowhead is also developing plozasiran for severe hypertriglyceridemia and dyslipidemia. Ascendis Eyes Approval in Rare Bone Growth Disorder Also awaiting a rare disease decision is Ascendis Pharma, which is advancing TransCon CNP, also known as navepegritide, for the treatment of children with achondroplasia. The FDA is set to release its verdict by Nov. 30. Achondroplasia is a rare, genetic disease characterized by dwarfism due to faulty bone development. Ascendis is targeting a specific disease type caused by mutations in the FGFR3 gene, resulting in manifestations in different tissues across the body. Aside from bone problems, patients with this specific type of achondroplasia also suffer from muscular, neurological and cardiorespiratory problems. Around 250,000 people worldwide are affected by this disease, according to Ascendis. TransCon CNP works by providing sustained exposure to the C-type natriuretic peptide, a molecule that helps stimulate bone growth and counter the inhibiting effects of FGFR3 mutations. Through this mechanism, Ascendis contends that its drug candidate could help ease the burden of symptoms and complications in achondroplasia. Ascendis tested the TransCon CNP in ACcomplisH, a randomized, double-blinded and placebo-controlled Phase II study that enrolled 42 patients who received 6 μg, 20 μg or 50 μg of the drug. Results, published in The Lancet in November 2023, showed that patients on TransCon CNP saw significant improvements in annualized growth velocity, alongside other key benefits, such as a stabilized quality of life and better physical functioning. In a June 2025 statement , Ascendis Chief Medical Officer Aimee Shu said the biotech’s clinical development program for TransCon CNP is the “first ever to demonstrate improvements beyond linear growth at 52 weeks compared to placebo.” Cytokinetics Could Challenge BMS in Cardiomyopathy Market Closing out this list is Cytokinetics and its cardio candidate aficamten, which is currently under FDA review for obstructive hypertrophic cardiomyopathy (oHCM). The FDA’s target decision date is Dec. 26. Aficamten blocks the cardiac myosin protein, which plays a role in heart contraction, allowing the oral drug candidate to address a key symptom of oHCM. Data from the Phase III MAPLE-HCM study, presented in August at the 2025 European Society of Cardiology Congress, demonstrated a significant improvement in exercise capacity over metoprolol, a beta-blocker used as standard-of-care in the disease. At the time, analysts at Truist Securities said aficamten’s results position it to be “potentially best-in-class” in oHCM. They called the MAPLE-HCM data “robust,” noting that outperforming metoprolol could help aficamten distinguish itself in the oHCM market. Cytokinetics filed for aficamten’s approval in late 2024, at which time the FDA provided an initial target action date of Sept. 26, 2025 . In May, however, the regulator extended its review by three months to review an additional risk mitigation strategy submission. If approved, aficamten will compete with BMS’s Camzyos, also a cardiac myosin inhibitor that won approval for oHCM in 2022 . The pharma is testing the drug in a non-obstructive form of the heart disease, but a readout in April this year was disappointing : Camzyos was unable to significantly outperform placebo in terms of exercise capacity outcomes, nor did it lead to better physical function.