Global biopharmaceutical company Mallinckrodt plc announced results of a retrospective, observational medical chart review subgroup analysis assessing real-world treatment outcomes among African Americans with advanced symptomatic sarcoidosis who initiated therapy with Acthar Gel (repository corticotropin injection).
One day after BridgeBio Pharma struck a nearly $1 billion oncology pact with Bristol Myers Squibb, the California-based company announced plans to sell a priority review voucher received in February.
Cortexyme, a company focusing on treatments for degenerative diseases, entered into a deal to acquire Novosteo, which plays in the rare disease space. Once the deal is finalized, the merged companies will operate under the name Quince Therapeutics and trade under the ticker symbol QNCX.
LogicBio Therapeutics was granted the green light to continue dosing participants in the company’s Phase I/II Sunrise trial investigating the safety of LB-001 in patients with methylmalonic acidemia (MMA), characterized by methylmalonyl-CoA mutase (MMUT) gene mutations.
The U.S. Food and Drug Administration granted full approval to Orphalan’s Cuvrior (trientine tetrahydrochloride) for the treatment of Wilson’s disease, a rare genetic disorder. The approval announcement marks the first new treatment available for patients in over five decades.
Pfizer Inc. said on April 28 the company would open the first U.S. trial sites for its experimental gene therapy for a muscle-wasting disorder, after the Food and Drug Administration lifted the regulatory agency’s hold on a late-stage study.
EVERSANA, the pioneer of next-generation commercial services to the global life sciences industry, and Vector Pharma FZCO of Dubai UAE, the leading full-service distributor for rare disease, oncology and highly specialized therapeutics in the Middle East, North Africa and Turkish markets, announced a strategic partnership to expand services across the Middle East and North African markets.
Takeda Pharmaceutical announced positive results from the Phase III SHP643-301 trial of Takhzyro (lanadelumab) in kids ages 2 to <12 years with hereditary angioedema (HAE). The drug is already approved for HAE in people 12 years and older.
Aeglea BioTherapeutics announced on April 11 promising Phase III results for patients diagnosed with arginase 1 deficiency (ARG1-D), a rare hereditary metabolic disorder. Aeglea said on April 12 the company had submitted its first-ever Biologics License Application (BLA) to the U.S. Food and Drug Administration for the drug.
It’s no longer science fiction. The use of drones promises to provide those with chronic medical conditions with faster and easier access to the therapies they need.