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

Drug Target Review

Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.

Disease 52
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FDA Approves Duvystat, New Oral Treatment for Duchenne Muscular Dystrophy (DMD)

PLOS: DNA Science

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. The gene presents a complex landscape.

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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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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] 2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1]

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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.

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FDA approves Nexviazyme® (avalglucosidase alfa-ngpt), an important new treatment option for late-onset Pompe disease

The Pharma Data

Approval is based on positive Phase 3 data demonstrating improvements in key disease burden measures and establishing its safety profile Nexviazyme specifically targets the M6P receptor, the key pathway for enzyme replacement therapy, to effectively clear glycogen build-up in muscle cells. LOPD symptoms may present at any age.

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Good things come in 3s

SugarCone Biotech

Over 70% of new patients are diagnosed with stage IV disease, also called extensive stage disease (ES-SCLC). The results were remarkable for this very sick patient population, with a median OS of 14 months at the dose of 10 mg given every 2 weeks: The data supported FDA approval, in May 2024, of tarlatamab for refractory ES-SCLC.