Tag Archive for: rare disease

The California pharma is building up to its first-ever approval with promising late-stage data for its once-daily investigational acromegaly pill paltusotine, an alternative to the injectable standard of care.

Seeking to deepen its neurology and rare disease pipelines, AstraZeneca’s Alexion has joined forces with Verge Genomics to leverage its artificial intelligence platform in drug discovery and development.

The case will be “withdrawn from adjudication” until Sept. 18, according to the filing, and this will allow the FTC to consider other avenues of resolving the matter, including a settlement.

After two prior setbacks, the regulator has finally approved Ipsen’s palovarotene to treat fibrodysplasia ossificans progressiva. It’s the first treatment for the ultra-rare bone disease.

Following its Scorpion Capital controversy in March, Harmony Biosciences is seeking to deepen its pipeline with an acquisition of Zynerba Pharmaceuticals’ cannabinoid therapies for neuropsychiatric disorders.

The market opportunity for pulmonary arterial hypertension drugs stretches well beyond the 500–1,000 Americans diagnosed with the disease each year—and biopharma is taking note.

The deal is the first large acquisition under new CEO Christopher Viehbacher as he seeks to return the drugmaker to growth.

Despite beating analyst expectations, Takeda sustains a mid-stage defeat in a rare genetic disorder, leaving the fate of an investigational enzyme replacement therapy uncertain.

The British company also said Sharon Barr, head of research and development at Alexion, would succeed Mene Pangalos, the long-time biopharmaceuticals head of research at AstraZeneca.

The program—a joint initiative between CDER and CBER—aims to accelerate the development of therapies for rare diseases.