Drugs

START Pilot Helps Rare Disease Treatments: Cavazzoni

CDER director Patrizia Cavazzoni says the Support for clinical Trials Advancing Rare disease Therapeutics (START) pilot program started by CDER and CBER is helping to accelerate the development of novel drugs and biologics. In an online post, Cavazzoni says the two Centers created START “with hopes that the insight gained through the pilot will provide information on how best to facilitate more efficient development of potentially life-saving therapies for rare diseases and help sponsors generate high-quality, actionable data to support future new drug or biologics license applications.”

The program is starting with three CDER and four CBER participants. They will have access to frequent advice from FDA staff to address product-specific development issues, including clinical study design, choice of control group, and fine-tuning the choice of patient population, Cavazzoni writes. The increased interactions are said to include addressing early development issues that may otherwise delay or prevent a promising novel product from progressing to the pivotal clinical trial stage and ensuring a clear understanding of the information and data needed to facilitate product development.

CDER pilot participants are Larimar Therapeutics (Friedreich’s ataxia), Calico Life Sciences (Vanishing White Matter disease), and Denali Therapeutics (mucopolysaccharidosis Type IIIA [Sanfilippo syndrome]). CBER participants are Grace Science (NGLY1 deficiency), Moderna TX (isolated methylmalonic acidemia due to complete or partial methylmalonyl-coenzyme A mutase deficiency), Myrtelle, Inc. (Canavan disease), and Neurogene, Inc. (Bett syndrome).

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