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

The Pharma Data

Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).

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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. According to Dr. Richard J.

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Sunvozertinib

New Drug Approvals

2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1] 5] In China, it was conditionally approved in 2023 for the treatment of NSCLC and full approval is contingent on results of phase 3 clinical trials. [6] Sunvozertinib CAS 2370013-12-8 DZD9008, 584.1 12 (3): 217–224. PMID 38101437.

FDA
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Good things come in 3s

SugarCone Biotech

Two large and randomized clinical trials, IMpower-133 and CASPIAN, showing statistically significant improvements in outcomes when chemotherapy was combined with atezolizumab or durvalumab, respectively, in the first-line treatment of ES-SCLC. This Phase 3 trial compared tarlatamab with chemotherapy as second-line treatment.

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Vamorolone

New Drug Approvals

Example 2: Synthesis of the present invention Scheme C: Route of Synthesis of Vamorolone from 8-DM Vamorolone was synthesized in three synthetic steps from commercially available 8-DM. Hoffman, Efficacy and safety of vamorolone vs placebo and prednisone among boys with duchenne muscular dystrophy: a randomized clinical trial, JAMA Neurol.

FDA