Regulatory
In January 2019, the U.S. Food and Drug Administration (FDA) updated a 2015 draft guidance on rare diseases.
Phase 1/2 pheNIX Trial Would Represent First PKU Gene Therapy to Enter the Clinic
The shares are trading on XETRA, the electronic trading platform of Deutsche Börse.
The government charged the three companies with using charitable foundations they financially supported to finance copays of Medicare and Medicaid patients, which is a violation of law.
The current Spinraza cost is $750,000 for the first year and $375,000 for every year after for the life of the patient. It is approved for all forms of SMA.
With the clock winding down on his tenure at the U.S. Food and Drug Administration, Commissioner Scott Gottlieb continues to take aim at a number of concerns he has raised over the past two years, including the dangers of vaping and unsanctioned use of stem cells.
Across the globe, pharma and biotech companies report deals and pipeline updates, with news from Boehringer Ingelheim, Metrion, Elvie, and more.
The CRL is not totally unexpected, given that last month the company received a multi-disciplinary review (DR) letter from the FDA regarding its New Drug Application.
The U.S. Food and Drug Administration (FDA) approved ADMA Biologics’ Asceniv to treat Primary Humoral Immunodeficiency Disease (PIDD or PI) in adults and adolescents.
Evotec SE announced that it has completed its conversion into a company under European law with its registration in the commercial register of the District Court of Hamburg.
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