Lenacapavir Sodium

Europe’s Committee for Medicinal Products for Human Use has reversed its decision on Eli Lilly's Alzheimer's disease treatment Kisunla, giving it a positive opinion in a limited patient population. Four months after giving Eli Lilly’s Alzheimer’s disease treatment a thumbs-down, Europe’s Committee for Medicinal Products for Human Use (CHMP) has recommended it for approval, albeit in a restricted patient population.The positive opinion covers Kisunla's use in patients with early Alzheimer’s symptoms who have one or no copies of the apolipoprotein E ε4 (ApoE4) gene. The drug is not recommended for individuals carrying two copies of the ApoE4 gene. Those patients are more susceptible to amyloid-related imaging abnormalities (ARIA), which are a primary side effect of currently approved Alzheimer’s drugs.The restriction is the same as the one applied by European regulators when it granted marketing authorization to Eisai and Biogen’s Alzheimer’s drug Leqembi three months ago. Those companies went through the same rejection/appeal process as Lilly did with European regulators.Lilly went through the same go-round with regulators in the U.S. before it secured approval of Kisunla in July of last year. Then, earlier this month, the FDA signed off on a new dosing regimen for the treatment, featuring a more gradual titration, which leads to a significantly lower rate of ARIA-related brain swelling. The new dosing regimen also applies to Thursday’s thumbs-up from the CHMP. Endorsement for Gilead’s PrEP injection  The CHMP has also provided a positive opinion on Gilead Sciences' HIV pre-exposure prophylaxis (PrEP) Yeytuo (lenacapavir), a twice-a-year subcutaneous injection which has been hailed as one of the most important breakthroughs in the effort to end the epidemic.The FDA blessed the shot last month under its commercial name Yeztugo.The CHMP recommendation applies to adults and adolescents and came under an accelerated pathway, “because it is considered to be of major public health interest in the EU and the rest of the world,” the regulator said.It was also evaluated under the EU-Medicines for all program, which included participation from the World Health Organization and regulators from several African and Asian nations. The program helps speed approvals where “regulatory capacity may be limited,” the CHMP said.“Lenacapavir for PrEP has the potential to become a critical tool for public health, helping to expand prevention options for people who face the highest barriers to care,” Dietmar Berger, M.D., Ph.D., Gilead’s chief medical officer, said in a release. Other CHMP decisions A month after Supernus Pharmaceuticals made a $561 million bet on Zurzuvae, buying out its developer Sage Therapeutics, the CHMP has registered a positive opinion on the treatment for postpartum depression. The Biogen-partnered treatment was endorsed by the FDA in August 2023, along with a rejection from the regulator in a much larger patient population, major depressive disorder.The European regulator has also signed off on rare disease specialist IntraBio’s Aqneursa for the treatment of the lysosomal storage disorder Niemann-Pick disease type C (NPC). The endorsement comes 10 months after the FDA approved two treatments for NPC in consecutive weeks, Aqneursa and Zevra Therapeutics’ Miplyffa, which has yet to win a nod in Europe.Less than three weeks after the FDA approved KalVista Pharmaceuticals' Ekterly for acute attacks of hereditary angioedema (HAE), the CHMP has followed suit. This is the first oral treatment for HAE recommended for approval in Europe, reducing the burden of injection on patients and speeding up the time between onset of attack and administration of the medicine, the CHMP said. Ono Pharmaceuticals has won a CHMP nod for Romvimza, for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT), a condition where the tissue surrounding the joints and tendons expands abnormally, forming outgrowths of the joint. The drug, which was acquired last year in Ono’s $2.4 billion buyout of Deciphera Pharmaceuticals, was endorsed by the FDA in February of this year, joining Daiichi Sankyo’s Turalio as the only other marketed treatment for TGCT.Ionis Pharmaceuticals' Tryngolza, for the treatment of adults with familial chylomicronemia syndrome, has gotten a thumbs-up from CHMP. The rare, autosomal recessive inherited disorder is characterized by elevated levels of triglycerides in the blood. The FDA approved the treatment in December of last year.Also receiving a CHMP nod is Servier’s Voranigo for low-grade astrocytoma or oligodendroglioma, two rare malignant brain tumors. The FDA blessed the treatment in August of last year.

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) landed on a number of high-profile decisions this week, including changing course on Eli Lilly’s Alzheimer’s drug in certain patients and recommending Gilead’s twice-yearly HIV preventive approach. Overall, CHMP said Friday that it is endorsing the authorization of 13 new medicines. It also said that the European Commission should reject three drugs, including Sarepta’s troubled Duchenne drug Elevidys , which Roche is licensed to distribute in Europe. Lilly’s Alzheimer’s drug Kisunla initially received a negative opinion in March. At the time, CHMP noted the benefits of the drug did not outweigh the risks of patients developing bleedings and swellings of the brain called amyloid-related imaging abnormalities (ARIA), which can be fatal. Kisunla was approved in the US in July 2024. CHMP has now reversed its previously negative opinion on Kisunla after reviewing Lilly’s new dosing plan, which includes starting off with a lower dose and additional measures to reduce the risk of ARIAs. Regulators have been wary of approving drugs with risks of ARIAs, such as Eisai and Biogen’s Alzheimer’s drug Leqembi, which was also initially rejected by the EMA last July. Since then, Leqembi was approved by the EC in April this year, becoming the first of its kind to be approved in Europe. It secured FDA approval in January. CHMP also endorsed Gilead’s twice-yearly injectable lenacapavir to prevent HIV. Lenacapavir is currently approved in Europe as a treatment for adults with multidrug-resistant HIV-1 infection, and not as a preventive measure. In a landmark move, lenacapavir was approved as PrEP in the US last month under the brand name Yeztugo. The same preventive product would have a different name in the EU : Yeytuo. Elsewhere, the European regulators also gave a positive opinion for Pfizer and BioNTech’s LP.8.1-adapted monovalent Covid-19 vaccine for individuals aged six months and older. This comes after the EMA’s Emergency Task Force recommended in May that Covid-19 vaccines need to be updated to target the LP.8.1 strain. Pfizer and BioNTech have already started manufacturing their LP.8.1-adapted Covid-19 vaccine. As soon as the EC gives its approval, the companies will be ready to ship the vaccine, they said in a Friday release . CHMP also recommended the approval of the following products: The EU regulators suggested recommending the following label expansions: CHMP recommended rejecting TETEC Tissue Engineering Technologies’ autologous cartilage-derived articular chondrocytes called Jelrix, as well as Prilenia Therapeutics’ Huntington’s disease drug Nurzigma.