Regulatory Roundup: Top News Stories – Week Ending August 31, 2017

I’m publishing my blog a day early due to the long holiday weekend, and wow this has been quite a week in regulatory news.  The FDA approved the first CAR-T therapy, along with two new novel drugs, a new biosimilar, and a couple of supplemental approvals for new indications.  The FDA also sent two refuse-to-file letters and granted three new breakthrough therapy designations.  Here is my recap of the top regulatory stories this week:

On Wednesday the FDA approved Novartis’s CAR-T therapy ushering in a new approach to treating cancer in a landmark decision for the rapidly advancing field of cell therapy.

The FDA has granted accelerated approval to Chemo Research SL’s benznidazole  for treatment of children aged 2 to 12 years with Chagas disease, becoming the first treatment approved in the United States for this rare disease.

The Medicines Company announced FDA approval of VABOMERE™ (meropenem and vaborbactam) for the treatment of adult patients with complicated urinary tract infections.

Boehringer Ingelheim received approval from the FDA for its Cyltezo, another biosimilar to AbbVie Inc.’s Humira inflammation product. This is the sixth copycat biologic to gain FDA approval and the second that references Humira.

The FDA approved Genentech’s Actemra for treatment of CAR T cell induced Cytokine Release Syndrome. Actemra was initially approved in 2010 for the treatment of moderate to severe rheumatoid arthritis, and has had supplemental approvals for systemic juvenile idiopathic arthritis and also for giant cell arteritis.

Novo Nordisk said that the FDA approved an expanded use of its diabetes drug Victoza to reduce the risk of cardiovascular events such as heart attack and stroke. Victoza was approved in 2010 to treat patients with type 2 diabetes.

Merck KGaA received approval from the EMA to market their multiple sclerosis drug Cladribine in Europe. The company will launch the pill first in the U.K. and Germany as “Mavenclad” and its executives are already talking about taking another pass at winning approval from the FDA.

The European Commission has approved Aveo’s lead drug tivozanib for advanced renal carcinoma.

Sunovion Pharmaceuticals has submitted a NDA to the FDA for dasotraline, a novel investigational, dual-acting dopamine and norepinephrine reuptake inhibitor (DNRI), for the treatment of children, adolescents and adults with attention deficit hyperactivity disorder (ADHD).

 Alkermes announced the initiation of its rolling submission of a NDA to the FDA for ALKS 5461, an investigational medicine for the treatment of major depressive disorder (MDD). The company expects to complete the submission of the NDA for this Fast Track designated medicine by year-end 2017.

Kyowa Hakko Kirin won FDA breakthrough therapy designation for their late stage lymphoma drug mogamulizumab:

The FDA has given Daiichi Sankyo’s DS-8201 for HER2-positive metastatic breast cancer a breakthrough therapy designation

Abeona Therapeutics also landed a breakthrough therapy designation for their Phase III-ready EB-101 gene therapy program for patients with Recessive Dystrophic Epidermolysis Bullosa.

FDA has accepted Genentech’s sBLA and granted priority review for Gazyva for previously untreated Follicular Lymphoma

Apricus Biosciences, Inc. filed a resubmission of its NDA for Vitaros™ (alprostadil, DDAIP.HCl) with the FDA. Apricus anticipates a six-month review by the FDA with a projected PDUFA goal date in the first quarter of 2018.  Vitaros is a novel, on-demand topical cream for the treatment of erectile dysfunction.

SCILEX Pharmaceuticals Inc. (a majority-owned subsidiary of Sorrento Therapeutics) resubmitted the NDA for its lead product candidate, ZTlido™ (lidocaine patch 1.8%).   ZTlido is a non-opioid, lidocaine patch currently in development for the relief of pain associated with post-herpetic neuralgia (“PHN”), a severe neuropathic pain condition.

Acorda Therapeutics said that the FDA has sent them a “refusal to file” letter for their investigational Parkinson’s drug “Inbrija”, an inhalable form of the long-used Parkinson’s treatment levodopa. Acorda stressed that the FDA’s concerns are “addressable” and that the agency hasn’t asked the company to run any new clinical trials.

The FDA also issued another refuse to file letter to SteadyMed Ltd. This pertains to their NDA for its pulmonary arterial hypertension (PAH) treatment Trevyent. In the letter, the agency said the NDA was “not sufficiently complete” and called for additional information about testing procedures for the product, among other things.

Bloomberg News reported that FDA warning letters are down significantly for the first seven months of 2017.  From January to July 2017, the agency sent 265 warning letters to companies, the lowest tally for the first seven months of any year since 2008:

FDA Commissioner Scott Gottlieb said that the agency will advance a new framework to better regulate stem cell therapies this fall. The new policies will be set via a series of guidance documents to “give current product developers a very reasonable period of time” to interact with FDA to determine if they need to submit a marketing application.

In other news

Gilead Sciences will buy Kite Pharma in a $11.9 billion deal, strengthening Gilead’s position in the emerging space of cell therapy.

Gene therapy firm Regenxbio has signed an agreement to acquire Dimension Therapeutics for $86 million.

Teikoku Pharma USA reported positive topline results from their Phase 2 clinical trial of TPU-006, non-opioid pain management patch

Novartis purchased Xoma’s failed PhIII cardiovascular disease drug gevokizumab for $31 million

Jazz Pharmaceuticals is teaming up with ImmunoGen on a slate of armed antibody programs.

Celgene made an additional investment in FORMA Therapeutics this week.  Looks like they are expanding their scope of research to include neurodegeneration, protein homeostasis and inflammation/immunology

AstraZeneca struck a $400 million Parkinson’s drug deal with Takeda. Under this agreement, AstraZeneca will lead Phase I testing of MEDI1341 while Takeda will lead future clinical development.

Despite earlier this month announcing it’s considering a sale of ADHD offerings, Shire has launched its latest Adderall formulation in the U.S.

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