Drugs

FDA Abandons Amicus Requirement for Added Study on Fabry Drug

FDA has abandoned a requirement imposed on Amicus Therapeutics to conduct an additional Phase 3 study to assess gastrointestinal symptoms with its oral precision medicine migalastat for Fabry disease, a move that now opens a path for an NDA filing in the fourth quarter seeking accelerated approval. The European Commission has granted full approval for migalastat under the trade name Galafold as a first line therapy for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease and who have an amenable mutation. Marketing applications have also been approved in Switzerland and Israel, and submissions are under review in Japan, Canada and Australia.

Amicus says it intends to base its NDA on existing data, including reduction in disease-causing substrate (GL-3), as well as the totality of data from completed clinical studies. “Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease and stroke,” it says. Migalastat works by “stabilizing the body’s own dysfunctional enzyme, so it can clear the accumulated disease substrate in patients who have amenable mutations,” it explains. “An amenable mutation is one that is responsive to therapy with migalastat based on a proprietary in vitro assay.”

Amicus CEO John Crowley told Forbes that the company convinced FDA to look at its data using new analyses, including some that had been asked for by European regulators, and patient testimony. “We didn’t ask them to lower the bar,” he says. “We just asked them to review the existing data.” Crowley is well known for developing a drug for Pompe disease from which his daughter suffers. Earlier this year, president Donald Trump highlighted her story in a speech to Congress as an example of the need for more flexibility at FDA.

FDA watchers are speculating that the arrival of new FDA commissioner Scott Gottlieb may have encouraged agency reviewers to help find a path forward for the drug. But Crowley told Forbes that he doesn’t think Trump’s speech nor Gottlieb’s appointment had any impact on the agency decision. He did argue, however, that there is “an emerging framework at the FDA for rare diseases.” He noted the approval of BioMarin's Bineura for Batten’s disease and an expanded approval for Vertex’s Kalydeco for cystic fibrosis as examples.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome