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Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

FDA Law Blog: Drug Discovery

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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FDA Programs to Support Advanced Manufacturing: Where Innovation Meets Regulation

The Premier Consulting Blog

It is recognized, however, that there is a learning curve across the industry and FDA that is inherent to the development of such novel technologies. To prevent this learning curve from becoming a roadblock on the path to regulatory approval, the FDA has developed several collaborative programs to support advanced manufacturing.

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FDA to use new review tool on Sarepta’s gene therapy work

BioPharma Drive: Drug Pricing

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene therapy applications it later brings to the regulator.

Therapies 195
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When is a Confirmatory Trial “Underway” or Conducted with “Due Diligence” Enough for Accelerated Approval? FDA Explains Its New Authorities

FDA Law Blog: Drug Discovery

FDAs withdrawal authority when a confirmatory trial is not conducted with due diligence was expanded to include that FDA could specify the conditions for a postapproval study. Considering FDAs new authority to specify the conditions for such confirmatory trials (e.g.,

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Gene therapy faces fresh uncertainty as two more top FDA officials depart

BioPharma Drive: Drug Pricing

The reported dismissal of high-ranking CBER officials Nicole Verdun and Rachael Anatol resurfaced lingering concerns about how gene therapies will be regulated under new FDA leadership.

Therapies 321
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The AI model that is changing clinical trial design

Drug Target Review

Unlearn’s early collaboration with regulators has helped it navigate this space effectively. The company worked closely with agencies including the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) from the outset, aligning its methodology with evolving guidance.

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FDA Approves Label Updates for Bristol Myers Squibb Cell Therapies, Removes REMS

The Pharma Data

FDA Approves Streamlined Monitoring Requirements and REMS Program Removal for Bristol Myers Squibb’s CAR T Cell Therapies Breyanzi and Abecma, Marking Milestone Toward Expanding Access to Cancer Treatment In a significant regulatory development, Bristol Myers Squibb announced that the U.S.