The U.S. Food and Drug Administration approved Pfizer Inc.’s supplemental Biologics License Application for Panzyga to treat adult patients with a rare neurological disease of the peripheral nerves called chronic inflammatory demyelinating polyneuropathy (CIDP).
Find out which biopharma companies are raking in the cash this week, as companies from around the globe provide updates on their financing rounds and IPOs.
Alnylam Pharmaceuticals presented complete results from the company’s ILLUMINATE-B Phase III study in children under 6 years with primary hyperoxaluria type 1 (PH1), a rare disorder affecting the kidneys.
U.K.-based Celleron Therapeutics signed a licensing agreement with Swiss pharma giant Roche for the worldwide rights to the cancer drug emactuzumab, a monoclonal antibody directed against colony-stimulating factor 1 (CSF-1R) expressed on macrophages.
David Fajgenbaum’s life changed forever one summer day in 2010. After experiencing fatigue, his liver, kidneys, bone marrow, heart, and lungs suddenly began to shut down. He was admitted to the intensive care unit where doctors found that he had a retinal hemorrhage that left him blind in his left eye. David drifted in and out of consciousness, was put on a feeding tube, and received blood transfusions. In weeks, the 26-year-old medical student sharply deteriorated and was given last rites.
The U.S. Food and Drug Administration approved Clinuvel Pharmaceuticals Ltd.’s treatment for a rare inherited disorder that causes skin damage from exposure to light, sending the Australian drugmaker’s shares to a record high.
Deep Genomics’ proprietary artificial intelligence-based drug discovery platform identified a novel treatment target and corresponding drug candidate for the rare and potentially life-threatening genetic disorder Wilson disease.
Genentech released full data from the company’s pivotal Phase III SAkuraStar trial of satralizumab as a monotherapy for NMOSD.
Sarepta Therapeutics Inc. was informed that an adverse event report was erroneously submitted to the U.S. health regulator regarding an ongoing study of the company’s gene therapy for Duchenne muscular dystrophy (DMD).
The U.S. Food and Drug Administration approved Daiichi Sankyo Co. Ltd.’s Turalio for adult patients with a type of rare, non-cancerous tumor affecting joints and limbs.