Tag Archive for: rare disease

The drug candidate, olezarsen, showed statistically significant reduction in levels of triglyceride compared to placebo, as well as showed a 100% reduction in inflammation of the pancreas.

In a settlement on September 1, the U.S. Federal Trade Commission cleared Amgen to proceed with its $27.8 billion acquisition of Horizon Therapeutics after several months of scrutiny. Some experts say the agreement is a setback for the agency.

Preston Taylor is a welcome addition to Greater Than One’s leadership team. According to Bindhu Gopalan, Chief Commercial Officer, “Preston’s strategic financial background, and his experience working with major agencies, will be essential to our efforts to better scale with clients as they grow”.

Nearly three years after European approval, the U.S. regulator has accepted Orchard Therapeutics’ BLA for its gene therapy OTL-200, being proposed for metachromatic leukodystrophy.

After nearly seven years, the company’s rare diseases arm Alexion has reached a settlement in an investors’ lawsuit over alleged unethical sales practices for its hemoglobinuria therapy Soliris.

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The case will be “withdrawn from adjudication” until Sept. 18, according to the filing, and this will allow the FTC to consider other avenues of resolving the matter, including a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