Top Regulatory Affairs News Stories – Week Ending September 8, 2017

Summer is over and there has been a flurry of new regulatory news this week including new approvals and clinical holds.  Also, there were a few new partnership announcements and a major layoff at Eli Lilly.  Here is my summary of the top news stories this week:

Pfizer has secured approval from the FDA for its Mylotarg (gemtuzumab ozogamicin) to treat adults with newly diagnosed CD33-positive acute myeloid leukemia (AML).

Durham-based Bioventus has received approval from the U.S. Food and Drug Administration for its treatment, Durolane.

 Merck has secured approval from the European Commission for its Keytruda (pembrolizumab) to treat certain patients with locally advanced or metastatic urothelial carcinoma (a type of bladder cancer).

Deciphera received FDA orphan drug designation for DCC-2618 to treat glioblastoma multiforme and anaplastic astrocytoma:

Bayer has submitted a BLA with to the FDA for its long-acting site-specifically PEGylated recombinant human factor VIII to treat Hemophilia A.

Verastem, Inc. plans to seek U.S. approval early next year for its lead blood cancer candidate duvelisib, based upon positive results from a Phase 3 study:

Regeneron and Sanofi received breakthrough status for their PD-1 program:

Cellectis reported that the FDA has put a clinical hold on two of its clinical trials for its gene-edited allogeneic CAR-T therapy following a patient death.

Worried by evidence of safety issues, the FDA ordered a halt to enrollment of new patients in Bristol-Myers’ studies for several combination drug programs for multiple myeloma.  In addition, a hold was placed on Celgene’s clinical study for a drug combination product in multiple myeloma patients.

This week the CDRH released eight warning letters for device manufacturers from the US, Brazil, the Netherlands, Germany, China and Bulgaria:

The FDA is reorganizing the way it handles manufacturing inspections, saying the new system should speed drug approvals and eliminate surprises that drugmakers sometimes face when plant questions delay them.

In other news…

Janssen Biotech, Inc. has broken off a development deal with Maryland biotech Macrogenics for an early-stage cancer candidate, shutting down a Phase 1 dose-escalation study after treatment-related neurotoxicity was observed in some patients.

Merck announced it was acquiring Munich-based Rigontec, a company focused on pioneering therapeutic access to the retinoic acid-inducible gene I (RIG-I) pathway, which part of the innate immune system.

Seattle Genetics announced that is has exercised its option to co-develop Genmab’s tisotumab vedotin, an antibody-drug conjugate (ADC) targeting tissue factor which is currently being evaluated in Phase I/II clinical studies in solid tumors.

Aptevo Therapeutics has agreed to sell its three marketed hyperimmune products to Saol Therapeutics for total consideration of up to $74.5m.

Insmed announced positive top line results from its Phase 3 study of Amikacin Liposome Inhalation Suspension in adult patients with Treatment-Refractory Nontuberculous Mycobacterial (NTM) Lung Disease.  Insmed plans to pursue accelerated approval based on the data from this study:

Alnylam has suspended dosing in trials of RNAi drug fitusiran after a patient with hemophilia A died in one of the studies.

Cytori Therapeutics is terminating half of its employees a little more than one month after its late-stage cell therapy to treat scleroderma failed to achieve statistical significance for both the primary and secondary trial endpoints.

Bioverativ announced a new partnership with Bicycle Therapeutics to identify new treatments for blood disorders.  This collaboration will use Bicycle’s technology program to identify bicyclic peptides that can treat hemophilia and sickle cell disease.

Eli Lilly said on Thursday it would lay off about 8 percent of its employees (3500 jobs) as they works to cut costs.

GSK will pay Adaptimmune up to $61 million to exclusively license the right to research, develop, and sell Adaptimmune’s so-called NY-ESO SPEAR T-cell therapy program.

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