Drugs

Avoid Tissue Agnostic Therapy Unintended Consequence: PhRMA

Pharmaceutical Research and Manufacturers of America (PhRMA) says that while it supports continued development of targeted therapies for molecularly defined conditions, it is important to avoid unintended consequences that might adversely affect orphan drug development. “If the incentives for development of products shift away from tumor histology to tissue agnostic treatments,” PhRMA says in a docket comment, “there could be a lack of investment in histological diseases with significant unmet medical needs. While addressing the molecular targets in different tumor types is important, it also will be important to continue to study drugs’ impact on histological targets. FDA policies should ensure incentives in both areas rather than favoring one over the other.”

The association also recommends that the agency consider a biomarker-positive, tissue-agnostic disease and a tissue-specific disease as the different diseases or conditions to maintain incentives to seek orphan drug exclusivity and orphan drug designation for both tissue-specific and biomarker-defined, tissue-agnostic diseases. And it says that FDA should exercise greater flexibility with respect to the evidence necessary to support orphan drug designation for a biomarker-defined tissue-agnostic disease.

The National Organization for Rare Disorders (NORD) says the current designation paradigm is flexible enough to appropriately address tissue agnostic therapies, orphan designation requests for oncology therapies should be comprehensive, and multiple exclusivity periods should not be awarded for the same drug in the same population.

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