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Working in Rare Diseases: A Business Model Suzanne Elvidge
February 28, 2025
Rare diseases, by definition, affect a small population.
The Curtain Has Risen on the Competition for the First Generic Drug, so What Other Innovative Drugs in the Rare Disease Field Can We Look Forward to? Xiaobin
January 23, 2025
The CDE website shows Qilu Pharmaceutical applied for the marketing of nusinersen generic. Once controversial for high price, it treats SMA. Now many firms are involved, and SMA treatment progresses.
Navigating the Landscape of Rare Diseases: Current Trends and Future Perspectives Neeta Ratanghayra
September 29, 2024
Rising Investment and Market Dynamics, Future Perspectives in the Orphan Drug Market and The Scenario in China.
Rare Disease Drug 101 - What are the Benefits to Apply for and Receive Orphan Drug Designation? Lin Zhang
June 29, 2021
There are many common diseases that affect millions of people across the world. On the other hand, there are so-called rare conditions that affect a small number of people.
UCB to buy rare disease therapies developer Zogenix for $1.9bn Pharmaceutical-Technology
January 21, 2022
The company’s subsidiary will be merged into Zogenix and the remaining shares of Zogenix common stock will be cancelled.
Pfizer partners with Beam Therapeutics to develop rare disease therapies ExpressPharma
January 11, 2022
Pfizer will collaborate with Beam Therapeutics in a deal worth as much as $1.35 billion to develop therapies for rare genetic diseases.
China increases accessibility to rare disease treatment NMPA/Xinhua
December 24, 2021
Two-and-half-year-old Xuanxuan survived a rare kidney disease in early December, thanks to fast-track approval of the transplant.
Recordati to acquire EUSA Pharma to expand rare disease products portfolio Pharmaceutical-Technology
December 06, 2021
Italian company Recordati has signed an agreement to acquire EUSA Pharma in a deal worth about $848.9m (€750m) to expand its rare disease products portfolio.
FDA, NIH, and 15 private organizations join forces to increase effective gene therapies for rare diseases WorldPharmaNews
October 29, 2021
The U.S. Food and Drug Administration, the National Institutes of Health, 10 pharmaceutical companies and five non-profit organizations have partnered to accelerate development of gene therapies...
FDA joins Bespoke Gene Therapy Consortium (BGTC) for rare diseases EuropeanPharmaceuticalReview
October 29, 2021
The US Food and Drug Administration (FDA), the National Institutes of Health (NIH), 10 pharmaceutical companies and five non-profit organisations have partnered to accelerate development of gene therapies for rare diseases.