Roche Holding AG’s Genentech Inc. unit received U.S. FDA approval on June 20 of Piasky (crovalimab) to treat adults and children 13 and older with paroxysmal nocturnal hemoglobinuria and a body weight of at least 40 kg (88 pounds).
Even though the U.S. FTC recently claimed a court victory in its campaign to shut down the listing of device patents for drugs in the FDA’s Orange Book, 80% of the listings targeted in the commission’s first round of warning letters remain in place more than seven months later.
Argenx SE gained U.S. FDA approval of subcutaneously given Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) for adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP). The company’s stock (NASDAQ:ARGX) was up 11.7% to $440.59 at the close of trading June 24. About 24,000 people in the U.S. are being treated for CIDP, and patients are generally diagnosed between 40 and 60 years of age.
In May, the U.S. FDA approved 11 new drugs, a significant drop from the 26 approvals in April, which was the sixth-highest monthly total since 2016. This also falls short of March’s record-setting 30 approvals, the highest number recorded by BioWorld.
Communication missteps, overreliance on a contract research organization and lack of clarity of U.S. FDA expectations for an expanded access protocol for emergency use resulted in a warning letter posted June 18 that highlights potential pitfalls for sponsor/investigators conducting the individualized studies.
Sarepta Therapeutics Inc. CEO Douglas Ingram said he expects “ferocious” demand for gene therapy Elevidys (delandistrogene moxeparvovec), granted full approval by the U.S. FDA for Duchenne muscular dystrophy (DMD). Shares of the Cambridge, Mass.-based firm closed June 21 at $16.72, up $37.22, or about 30% on the news.
The African Vaccine Manufacturing Accelerator is up and running, with more than $1 billion already pledged to the effort that’s aimed at supporting the sustainable growth of Africa's manufacturing base and contributing to the African Union's goal of producing 60% of the vaccines required by African countries by 2040.
Now that it has some experience with interchangeable biosimilars under its belt, the U.S. FDA is proposing an update to its interchangeability guidance that could eliminate the need for switching studies in some instances.
A nearly two-year-old partial clinical hold has been lifted by the U.S. FDA on PTC Therapeutics Inc.’s pivotal phase II study in Huntington’s disease. The agency had paused enrollment in October 2022, saying it wanted more data on PTC-518, an orally bioavailable small-molecule splicing modifier, before enrollment could continue.
China’s National Medical Products Administration (NMPA) approved Dizal Pharmaceutical Co. Ltd.’s golidocitinib for treating adults with relapsed or refractory peripheral T-cell lymphoma whose disease has progressed or was refractory to at least one prior systemic therapy.
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