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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.

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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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Jazz Pharma Unveils Late-Stage Xywav® Data in Narcolepsy at SLEEP 2025

The Pharma Data

The data were featured as part of four oral presentations selected by the Associated Professional Sleep Societies (APSS) for their scientific innovation and clinical relevance, with Jazz accounting for all the industry-sponsored late-breaking oral presentations at the meeting. Notably, the safety profile remained favorable.

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Biogen Advances Investigational SMA Therapy to Registrational Trials After Positive Phase 1 Data

The Pharma Data

Designed using the same fundamental mechanism as Biogen’s approved SMA treatment SPINRAZA (nusinersen), salanersen represents a next-generation approach aimed at greater potency and less frequent dosing—potentially requiring only once-yearly administration. Treatment-naïve individuals, to evaluate efficacy as a first-line therapy.

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Automated red blood cell exchange: bridging treatment gaps in sickle cell disease care

Drug Target Review

Dr Aaron Haubner, Senior Manager of North America Medical Affairs and Market Access at Terumo Blood and Cell Technologies , reveals that while promising new treatments emerge, urgent partnerships are needed to ensure this essential blood therapy reaches the patients who need it most.

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Sunvozertinib

New Drug Approvals

Sunvozertinib , sold under the brand name 舒沃哲, among others is an anti-cancer medication used for the treatment of non-small-cell lung cancer. [2] 2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1] 2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1]

FDA
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Good things come in 3s

SugarCone Biotech

Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis. Toxicities were challenging enough to cause 13% of patients to reduce dose or skip doses and 3% to discontinue treatment. months (versus 20% ORR and 8.3