Bionest

First Up For The New FDA RMAT Designation: Gene Therapeutics for Hemophilia

We recently wrote about the U.S. Food and Drug Administration’s (FDA) creation of a Regenerative Medicine Advanced Therapy (RMAT) designation in an effort to speed up the approval process for certain types of treatments, including gene therapies. The agency has specifically chosen gene therapy for hemophilia as its first focus, due to the considerable effort...

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Creating a New Global Pharmaceutical Giant: Takeda to Acquire Shire

The fifth try was the charm for Takeda Pharmaceuticals. On May 8, Takeda announced that their fifth offer to acquire Shire Pharmaceuticals had been accepted, subject to regulatory and shareholder approvals. This is the largest deal yet in 2018, and the largest ever for a Japanese pharmaceutical firm. If successfully completed as planned in the first half...

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Embryonic Stem Cell Technology and FDA

Stem cell research has elicited both excitement and controversy since its earliest days. Hematopoietic stem cell transplants have been employed for some time in the treatment of certain blood cancers. More recently, researchers have reported clinical successes in using bone marrow transplants to treat sickle cell disease as well as multiple sclerosis, where bone marrow...

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Vertex Under Fire

The Institute for Clinical & Economic Review (ICER) recently ruled that all three of Vertex’s cystic fibrosis (CF) drugs are considerably overpriced. ICER analyzed the prices for Kalydeco, Orkambi, and new treatment Symdeko based on a metric called “quality-adjusted life year,” which factors in both the quantity and the quality of life lived. While ICER...