Rare Disease– tag –
-
Oncology Drug Approval News Flash: FDA Grants Accelerated Approval to Dordaviprone (Modeyso) for H3 K27M-Mutant Diffuse Midline Glioma
On August 6, 2025, the U.S. Food and Drug Administration (FDA) granted accelerated approval to dordaviprone (Modeyso, Jazz Pharmaceuticals), a protease activator, for adult and pediatric patients (≥1 year) with progressive H3 K27M-mutant... -
Rare & Pediatric Disease News Vol.4: Organoid-Based Discovery for UBA5-Linked Pediatric Epileptic Encephalopathy
Screenshot Human Brain Organoids Reveal Therapeutic Potential for Rare Pediatric Epilepsy In June 2025, a research team led by Dr. Heather Mefford at St. Jude Children’s Research Hospital reported a groundbreaking study using human brain... -
Biotech Investment News: Major Pharmaceutical Deals of Astellas Pharma (2015–2025)
Strategic Review: Investing in Marketed Products and Next-Gen Modalities Astellas Pharma has executed a series of acquisitions and collaborations from 2015 to 2025 focused on immuno-oncology, ophthalmology, neurology, and gene therapy. K... -
Biotech Investment News: Pfizer’s Major Deals Over the Last Decade (2015–2025)
Strategic Review: From mRNA Success to Diversification in Rare Diseases, ADCs, and RNA Editing Pfizer strengthened its biopharmaceutical base with the acquisition of Hospira and GSK’s vaccine assets in 2015, followed by a major expansion... -
2025 Therapeutics Trend
What are the trends in therapeutics and market launch drugs in 2025? Market Expansion and Contested Competition for Obesity Drugs ADC Wave and Next Generation ADC (Antibody Drug Conjugate) Challenges for Rare Disease Therapeutic Roles an...
1