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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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Challenges in Clinical Development of Products for Rare Disease

thought leadership

Rare diseases are defined in the Orphan Drug Act as diseases or conditions that affect less than 200,000 people in the United States. Evaluating and developing treatments for rare diseases remains a key priority for FDA and is incentivized through the Orphan Drug Act.

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U.S. FDA Grants Orphan Drug Designation to Riliprubart for Treating Antibody-Mediated Rejection in Solid Organ Transplant Recipients

The Pharma Data

FDA Orphan Drug Designation for Antibody-Mediated Rejection in Solid Organ Transplantation The U.S. The announcement is particularly relevant given the lack of FDA-approved treatment options for AMR, a complication that poses a critical threat to transplant success and long-term graft survival.

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Good things come in 3s

SugarCone Biotech

Tarlatamab triggered an ORR of ~40% in refractory (second-line or later) SCLC patients in the DeLLphi-301 clinical trial published in 2023. The tarlatamab results have of course triggered a stampede of development of DLL3-targeted therapies. The Next Wave: Targeting B7H3 – a drug development tsunami in the making?

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FDA Approves

The Pharma Data

Food and Drug Administration (FDA) for the treatment of adult patients with deleterious or suspected deleterious BRCA -mutated ( BRCA m) metastatic castration-resistant prostate cancer (mCRPC). Patients should be selected for therapy based on an FDA-approved companion diagnostic for LYNPARZA. In the U.S., For the U.S.

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Metabolism of 2022 FDA approved small molecule drugs PART 1

Metabolite Tales Blog

Metabolism of 2022 FDA approved small molecule drugs – Part 1 Does CYP3A4 still rule? By Julia Shanu-Wilson It won’t come as much surprise to learn that of the 17 small molecules* approved by the FDA in 2022, CYP3A4 was the major player in drug metabolism.

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Metabolism of 2022 FDA approved small molecule drugs PART 2

Metabolite Tales Blog

Metabolism of 2022 FDA approved small molecule drugs part 2 Mixing it Up By Julia Shanu-Wilson In Part 1 of this topic we looked at metabolism of the small molecule drugs approved by the FDA in 2022 that were mediated by CYP3A4.