Despite an FDA advisory committee’s narrow ruling that Provention Bio Inc.’s teplizumab has benefits that outweigh the risks in treating diabetes, the agency sent the company a complete response letter (CRL) regarding the diabetes therapy’s priority BLA. The CRL came the evening of July 2, Provention said, the PDUFA date that had been set months before.
The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted 10-7 May 27 that the benefits of Provention Bio Inc.’s teplizumab outweighed its risks, but the vote was not a ringing endorsement of the anti-CD3 monoclonal antibody biologic that could be the first disease-modifying treatment for people at risk of developing type 1 diabetes (T1D).
Provention Bio Inc.’s diabetes candidate, teplizumab, is heading into a May 27 advisory committee meeting with a skinnier label than originally planned, which could signal a smoother path to approval. The positive briefing document the FDA put out Tuesday also suggested an approval path for what could be the first disease-modifying treatment available for type 1 diabetes (T1D).
If the FDA follows the advice of its Cellular, Tissue and Gene Therapies Advisory Committee, the U.S. could soon see its first approved islet transplant therapy, but few expect it to be broadly used. The adcom voted 14-9 April 15, with one abstention, that Celltrans Inc.’s donislecel, or cadaveric allogenic pancreatic islet cells, has an overall favorable risk-benefit profile for some patients with type 1 diabetes.
If the FDA follows the advice of its Cellular, Tissue and Gene Therapies Advisory Committee, the U.S. could soon see its first approved islet transplant therapy, but few expect it to be broadly used. The adcom voted 14-9 April 15, with one abstention, that Celltrans Inc.’s donislecel, or cadaveric allogenic pancreatic islet cells, has an overall favorable risk-benefit profile for some patients with type 1 diabetes.
The FDA told Provention Bio Inc. that its BLA under priority review for teplizumab needs some work but the May 27 advisory committee meeting to discuss the application in full is still on the calendar.
“We’re launching at the right time,” said Frank Sanders, president of Zealand Pharma A/S’s business in the U.S., in the wake of clearance granted by the FDA for the Copenhagen, Denmark-based firm’s glucagon analogue Zegalogue (dasiglucagon) in severe hypoglycemia.
Jaguar Gene Therapy LLC, a startup reuniting former Avexis Inc. executives to develop a portfolio of potential treatments for severe genetic diseases, announced its public debut Feb. 25 with more than $40 million in series A financing from co-creator Deerfield Management.
DUBLIN – Imcyse SA has successfully parlayed a research collaboration it entered with Pfizer Inc. in 2017 into a license agreement, under which it will finalize preclinical development of a drug candidate for rheumatoid arthritis (RA) based on its Imotope technology in return for an undisclosed up-front payment, equity investment and up to $180 million in milestones, as well as tiered royalties on product sales.
Veralox Therapeutics Inc., a Maryland-based startup developing a small molecule for halting aberrant platelet activation and thrombosis, said the FDA has granted the candidate, VLX-1005, orphan drug status for the treatment of heparin-induced thrombocytopenia (HIT).
