Top Regulatory News Stories – Week Ending September 6, 2019

Two decades after launching clinical trials, Kyowa Hakko Kirin finally received FDA approval for add-on Parkinson’s therapy

Mylan won tentative US approval for generic version of Lilly’s Alimta, but it could be years before it can be sold.

CARsgen Therapeutics Inc. announced the FDA has granted orphan drug designation to its investigational CAR-T cell therapy fully human anti-BCMA (B Cell Maturation Antigen) autologous chimeric antigen receptor (CAR) T Cells (ct053) for the treatment of multiple myeloma.

GeneTx Biotherapeutics and Ultragenyx Pharmaceuticals announced that the FDA has granted Orphan Drug Designation and Rare Pediatric Disease Designation to GTX-102 for the treatment of Angelman Syndrome.

Puma Biotechnology announced that the FDA has granted Orphan Drug Designation to NERLYNX® (neratinib) for the treatment of breast cancer patients with brain metastases.

Emalex Biosciences announced that it has received Fast Track designation for its investigational product, ecopipam, from the FDA for the treatment of patients with Tourette Syndrome (TS).

Ra Pharmaceuticals announced that the FDA granted Orphan Drug Designation to zilucoplan for the treatment of myasthenia gravis.

BioXcel Therapeutics announced that the FDA has granted Orphan Drug Designation for BXCL701, an investigational orally-available systemic innate immunity activator with dual mechanisms of action, for the treatment of Acute Myeloid Leukemia (“AML”).

In other news…

Vertex is paying $950 million for stem cell diabetes player Semma Therapeutics

Mallinckrodt is preparing for a possible bankruptcy amid a maelstrom of opioid claims.

Roivant’s Vivek Ramaswamy is closing in on a $3B cash deal to sell control of five Vant companies to Sumitomo Dainippon

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