“We’re going to battle,” PTC Therapeutics Inc. CEO Matthew Klein said, responding to a surprise negative opinion from the EMA’s Committee for Medicinal Products for Human Use on converting the conditional marketing authorization to full status for Translarna (ataluren) in the treatment of nonsense mutation Duchenne muscular dystrophy. The opinion applies to the renewal of the existing conditional authorization, too.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Alnylam, Dizal, Hemogenyx, Innocare, Neurocrine, Oncopeptides, Oscotec.
Insightec Ltd. broadened its CE mark approval for the Exablate Neuro, a focused ultrasound platform which treats essential tremors, to allow patients to have their second side treated. With some 60 million people estimated to be affected by essential tremor globally, Insightec hopes that with both sides treated, patients will have full body relief from tremor and therefore be able to resume everyday activities.
The U.S. FDA has broken out of the summer guidance doldrums in fine form, inking a series of nine draft and final guidances in the first half of September alone. The latest bolus includes a revised version of a guidance for the breakthrough devices program and two draft guidances for devices for weight loss, giving industry plenty to mull over as the final days of fiscal year 2023 trickle away.
Regulatory snapshots, including global submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Smartcardia.
The FDA has cleared an IND application for ADEL-Y01, being jointly developed by Oscotec Inc. and Adel Inc., for the treatment of Alzheimer’s disease. A phase Ia/b study will include healthy volunteers, and participants with mild cognitive impairment due to Alzheimer’s disease or mild Alzheimer’s disease.
The U.S. FTC put brand drug companies on notice Sept. 14 when the commissioners unanimously voted, 3-0, to issue a policy statement recognizing that improperly listed patents in the FDA’s Orange Book “may constitute an unfair method of competition.”
Supporting their conclusions with data from the same phase III study, the EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion for extending the use of Oncopeptides AB’s Pepaxti (melflufen) to earlier lines of treating relapsed, refractory multiple myeloma even as the FDA dug in its heels about withdrawing the drug from the U.S. market.
A U.S. FDA advisory committee’s backing keeps Alnylam Pharmaceuticals Inc.’s Onpattro (patisiran) on the road to a supplemental approval in treating a rare heart disease, but it couldn’t stop the company stock from sliding. Shares (NASDAQ:ALNY) closed Sept. 14 down 8.8% at $193.06, the day after the Cardiovascular and Renal Drugs Advisory Committee voted 9-3 that patisiran’s benefits outweigh the risks in treating cardiomyopathy of transthyretin-mediated amyloidosis.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Astellas, Aston, Bioray, Madrigal, Moderna, Morphosys, Nurexone, Rocket, Takeda.
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