Top Regulatory News Stories – Week Ending April 20, 2018

Rigel Pharmaceuticals announced that the FDA approved TAVALISSE™ (fostamatinib disodium hexahydrate) for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia who have had an insufficient response to a previous treatment.

Ultragenyx and Kyowa Kirin announced FDA approval of Crysvita® (burosumab-twza) for the treatment of children and adults with X–Linked Hypophosphatemia

Bristol-Myers Squibb announced that Opdivo (nivolumab) plus Yervoy (ipilimumab) (injections for intravenous use) was approved by the FDA as the first Immuno-Oncology combination therapy for previously untreated patients with intermediate- and poor-risk advanced renal cell carcinoma.

In a related story, the FDA has also granted a priority review to BMS’s sBLA for Opdivo (nivolumab) for the treatment of patients with small cell lung cancer with disease progression following 2 or more lines of therapy.

In a stunning about face, the FDA is now welcoming the NDA from Alkermes which it rejected two weeks ago.  This comes after the company clarified certain aspects of the NDA submission while no additional data or analyses were submitted.

GW Pharmaceuticals’ lead cannabis-based drug picked up a clear endorsement from regulators at CDER.

Solid Bio has cleared a partial hold, but its Duchenne muscular dystrophy gene therapy trial is still on hold for safety concerns

FDA Commissioner Scott Gottlieb told a House Appropriations subcommittee that the agency plans to “sharply increase” its release of disease-focused guidance documents to help drugmakers develop new treatments.


In other news…

After weeks of rumors, Takeda has made an official $60 billion bid to acquire Shire which was rejected.  Today Takeda announced an improved offer

Shire announced an agreement to divest its oncology business to Servier for $2.4 billion in cash.

Arbutus Biopharma and Roivant Sciences announced that they have entered into an agreement to launch Genevant Sciences, a jointly-owned company focused on the discovery, development, and commercialization of a broad range of RNA-based therapeutics.

Seattle-based Kineta Chronic Pain signed a $359 million exclusive option and license deal with Genentech to develop Kineta’s alpha9/alpha10 nicotinic acetylcholine receptor antagonists.

With its lead “monoclonal microbial” about to be tested for the first time on a human, Evelo Biosciences believes that this is the right time to float a $100 million IPO.

Catabasis Pharmaceuticals is laying off 21 employees, or 42% of its staff, in a restructuring that will throw its resources behind its lead asset, the Duchenne muscular dystrophy drug edasalonexent.

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