U.S. biotechs and regulators ushered in the era of gene therapy in 2023, experts at Bio Japan said, but medical reform is needed to pave the way for the “year of cell therapy” in 2024 and implement wider access to ultra-expensive cell and gene therapies.
With two complete response letters in the rearview mirror, Abbvie Inc.’s Vyalev (foscarbidopa/foslevodopa) has been approved by the U.S. FDA for treating Parkinson’s disease. The drug is the first subcutaneous 24-hour infusion of levodopa-based therapy for treating motor fluctuations in adults with advanced disease.
Clarity Pharmaceuticals Ltd. will begin early next year its pivotal phase III trial for its copper-based radiopharmaceutical, 64Cu-SAR-bisPSMA, for diagnosing prostate cancer in patients with biochemical recurrence following positive U.S. FDA feedback.
Shares of Novavax Inc. (NASDAQ:NVAX) dropped nearly 20% Oct. 16 to close at $10.15 after the company reported a serious adverse event had prompted a U.S. FDA clinical hold for its COVID-19-influenza combination and standalone flu vaccine candidates.
Coming on the heels of an advisory committee in which the U.S. FDA and its independent advisers grappled with trying to fit an ultra-rare disease development program into the confines of the agency’s “significant evidence” requirements, an Oct. 16 public meeting on a Rare Disease Innovation Hub the agency is setting up seemed like a welcome step in the right direction for rare disease patients, their caregivers and companies working in the space.
The U.S. FDA has approved the second hemophilia drug in nearly six months from Pfizer Inc. This one, Hympavzi (marstacimab), is for preventing or reducing bleeding in those age 12 and older with hemophilia A and B. Hympavzi heralds a couple of market boundary breakers: it’s the first and only anti-tissue factor pathway inhibitor approved in the U.S. for hemophilia A or B and the first hemophilia medicine approved in the U.S. to be administered using a pre-filled, auto-injector pen.
Genentech Inc. didn’t need to wait until Thanksgiving for the U.S. FDA to make up its mind. More than a month ahead of its PDUFA date, the agency approved the firm’s first-line breast cancer treatment, Itovebi (inavolisib), providing the oral therapy a place with other niched therapies from Astrazeneca plc and Novartis AG. Itovebi is to be combined with Pfizer Inc.’s palbociclib (Ibrance) and Faslodex (fulvestrant, Astrazeneca) for adults with endocrine-resistant, PIK3CA-mutated, hormone receptor (HR)-positive, HER2-negative, locally advanced or metastatic breast cancer.
“This was worse than our national election,” Eric Peterson said as he explained his vote Oct. 10 concluding that Stealth Biotherapeutics Inc.’s elamipretide is effective in treating Barth syndrome, an ultra-rare mitochondrial disease that currently affects 129 males in the U.S. Peterson, a vice provost, senior associate dean and professor at the University of Texas Southwestern Medical Center, was one of 10 members of the U.S. FDA’s Cardiovascular and Renal Drugs Advisory Committee (CRDAC) who came to that conclusion. Six others had a different opinion. Regardless of which way they voted, the panelists attested to how difficult the decision was.
The U.S. FDA has issued yet another complete response letter (CRL) for dasiglucagon, a glucagon receptor agonist, being developed by Zealand Pharma A/S for treating congenital hyperinsulinism, an ultra-rare disease that is also being targeted by at least two other companies. This CRL is pegged to the timing of a third-party manufacturing facility reinspection that was done in August and September. The agency also wants some additional clinical analysis from the phase III study.
Stealth Biotherapeutics Inc. had hoped the U.S. FDA would have approved its lead candidate, elamipretide, as the first treatment for Barth syndrome by now. Instead, it’s headed to a meet-up with the agency’s Cardiovascular and Renal Drugs Advisory Committee (CRDAC) Oct. 10. The discussion and vote at that meeting could be make-or-break for patients with the ultra-rare debilitating mitochondrial disease that has no approved therapies. “Barring support from CRDAC, the future of elamipretide for Barth syndrome in the U.S. is tenuous," Stealth CEO Reenie McCarthy told BioWorld.
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