The FDA is evaluating the efficacy of oral phenylephrine, a common ingredient in over-the-counter nasal decongestants, and may remove it from its non-prescription drugs guidelines (OTC Monograph). This decision follows an advisory committee's unanimous vote that current data do not support its efficacy as a nasal decongestant. The FDA emphasized that this proposed order is due to lack of efficacy, not because of safety. Public comments are open until May 7, 2025, before a final decision is made.
Regulatory Decisions for Novel Therapies
Vtama Review Extended for Atopic Dermatitis: The FDA has extended the review period for Organon's Vtama (tapinarof) cream for atopic dermatitis by three months, with a new target date of March 12, 2025. Vtama is already approved for plaque psoriasis.
Scemblix Approved for Newly Diagnosed CML: Novartis' Scemblix (asciminib) has received expanded FDA approval for the treatment of newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ PML). Scemblix is a STAMP inhibitor previously approved for advanced cases of Ph+ PML.
Orlynvah Approved for Uncomplicated UTIs: Iterum Therapeutics' antibiotic Orlynvah (sulopenem) has been approved by the FDA for the treatment of uncomplicated urinary tract infections in adult women with limited oral antibacterial treatment options. The drug is administered orally twice daily for five days.
Vyloy Approved for Gastric Cancer: Astellas Pharma's Vyloy (zolbetuximab) has gained FDA approval for the treatment of gastric or gastroesophageal junction adenocarcinoma. It is the first FDA-approved drug targeting the claudin 18.2 (CLDN18.2) protein in cancer. The approval is specifically for patients whose cancer is HER2-negative.
Avacincaptad Pegol Application Withdrawn in Europe: Astellas withdrew its European Medicines Agency application for geographic atrophy drug avacincaptad pegol, marketed as Izervay in the U.S., following discussions with the agency's Committee for Medicinal Products for Human Use.
Abrysvo Expanded to Include Adults 18-59: Pfizer's respiratory syncytial virus vaccine Abrysvo has expanded its FDA approval to include adults aged 18 through 59, broadening the market for the vaccine. It was initially approved for adults 60 and older and for maternal use during pregnancy.
Trodelvy Withdrawn for Urothelial Carcinoma: Gilead Sciences will no longer offer Trodelvy in the U.S. for treating urothelial carcinoma after discussions with the FDA. The drug had received accelerated approval in 2021, but a confirmatory Phase 3 trial failed.
Vyalev Approved for Parkinson's Disease: AbbVie has received FDA approval for Vyalev (foscarbidopa and foslevodopa), a drug/device combination for advanced Parkinson's disease, providing 24-hour subcutaneous infusion of the medications.
Hympavzi Approved for Hemophilia A and B: The FDA approved Pfizer's marstacimab, to be marketed as Hympavzi, for preventing or reducing the frequency of bleeding episodes in patients 12 and older with hemophilia A and B. It is administered as a once-weekly injectable medication.
Ocaliva Review Extended: The FDA needs more time to review Intercept Pharmaceuticals’ application seeking full regulatory approval of Ocaliva for primary biliary cholangitis.
Leqembi Faces Regulatory Obstacles in Australia: Eisai's Alzheimer's disease drug Leqembi faced a regulatory setback in Australia, with the Therapeutic Goods Administration deciding against registering the medication.
Companion Diagnostic Approved for Voranigo: The FDA approved a Thermo Fisher Scientific companion diagnostic for Servier Pharmaceuticals’ Voranigo (vorasidenib) for treating glioma patients with IDH1 or IDH2 mutations.
Optune Lua Approved for Non-Small Cell Lung Cancer: Novocure’s Optune Lua, a wearable device that kills cancer cells with electrical signals, has been approved for advanced cases of non-small cell lung cancer.
Dasiglucagon Rejected for Congenital Hyperinsulinism: Zealand Pharma’s dasiglucagon, a drug developed to prevent and treat hypoglycemia in children with congenital hyperinsulinism, was rejected by the FDA due to issues at a third-party manufacturer.
Advisory Committee Outcomes
Sotagliflozin Recommendation: An FDA advisory committee voted against the benefits of Lexicon Pharmaceuticals’ sotagliflozin outweighing its risks when used as an adjunct to insulin for adults with type 1 diabetes and chronic kidney disease.
Translarna Rejection Confirmed: The European Medicine Agency’s Committee for Medicinal Products for Human Use recommended against renewing the marketing authorization for PTC Therapeutics’ Translarna for Duchenne muscular dystrophy.
Elamipretide Vote: An FDA advisory committee voted that Stealth BioTherapeutics’ elamipretide was effective at treating Barth syndrome.
COVID-19 Updates
Nuvaxovid Authorized in Europe: The European Commission granted marketing authorization to Novavax’s updated COVID-19 vaccine, Nuvaxovid, for those aged 12 and older.
Clinical Hold on Combination Vaccine: The FDA pressed pause on Novavax’s plans for late-stage testing of a combination flu/COVID-19 vaccine due to a report of motor neuropathy in a single patient.
At-Home COVID-19 and Flu Test Authorized: The FDA granted marketing authorization to a Healgen Scientific over-the-counter, at-home test for both COVID-19 and influenza.
