Regulatory Roundup: Top News Stories – Week Ending September 29, 2017

The FDA approved Eli Lilly’s Verzenio (abemaciclib) to treat adult patients who breast cancer that has progressed following prior treatment:

Allergan received a refuse-to-file letter from the FDA for its sNDA to add an additional indication to its already-approved drug Vraylar (cariprazine).

Janssen received a complete response letter from the FDA for its rheumatoid arthritis candidate sirukumab.  In its rejection letter, the FDA requested additional clinical data so as to re-evaluate the safety of the drug in this indication.

Intarcia Therapeutics reported that the FDA has rejected its type 2 diabetes drug-device combo ITCA 650, sending the Boston-based company a complete response letter.

Based on FDA feedback at a recent guidance meeting, Novan says it will need to conduct a new pivotal study to support a U.S. marketing application for topical gel acne candidate SB204.

Dova Pharmaceuticals announced the submission of a NDA to the FDA for avatrombopag, a second generation orally administered thrombopoietin receptor agonist (TPO-RA) for the treatment of thrombocytopenia in patients with chronic liver disease who are scheduled to undergo a procedure.

Cascadian Therapeutics announced that the FDA has granted orphan drug designation to tucatinib for the treatment of HER2-positive (HER2+) metastatic colorectal cancer.

Audentes Therapeutics reported that the FDA has granted Rare Pediatric Disease and Fast Track designations for AT132, the company’s gene therapy product candidate being developed to treat X-Linked Myotubular Myopathy (XLMTM).

La Jolla Pharmaceutical Company announced that the EMA’s Committee for Medicinal Products for Human Use (CHMP) has issued favorable Scientific Advice regarding the EU regulatory pathway for LJPC-501 (angiotensin II) for the treatment of hypotension in adults with distributive or vasodilatory shock. Based on this advice, La Jolla intends to submit a MAA for LJPC-501 in the third quarter of 2018.

In other news…

Cambridge cancer drug startup Cue Biopharma is eying a $40 million IPO eight months after its launch:

Genocea Biosciences says it is shelving work on its lead, Phase III-ready program for genital herpes and is putting it up for auction.  In addition they are cutting 40% of its staff and refocusing the company towards Immuno-oncology and the development of neoantigen cancer vaccines.

Axovant’s experimental Alzheimer’s drug, intepirdine, failed to halt patients’ declines in cognition or improve their ability to function in daily life in a large phase 3 clinical trial.

 Aegerion Pharmaceuticals has finalized a $40 million settlement to resolve civil and criminal charges over its marketing of a cholesterol drug.

Pfizer has spun off four investigational drugs into a new medicines company dubbed SpringWorks Therapeutics.

Mateon Therapeutics announced Wednesday morning that it has halted its lead clinical trial due to lack of efficacy.

Nightstar Therapeutics, a clinical-stage gene therapy company developing treatments for rare inherited retinal diseases, announced the pricing of its initial public offering

AbbVie and Amgen have agreed to a deal in which Amgen won’t start selling Amjevita, a cheaper alternative to Abbvie’s blockbuster drug Humira, in the U.S. until 2023. All litigation between the two firms over the drugs also will be dropped.

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