Tag Archive for: rare disease

The deal is aimed at speeding up the development of therapeutics in areas including oncology, immunology and rare diseases.

Treatment with the drug candidate, acquired as part of the purchase of U.S. biotech firm Chinook for up to $3.5 billion, resulted in a meaningful improvement in proteinuria in patients suffering from IgA nephropathy when compared to placebo, the Swiss drugmaker said in a statement.

The label for the achondroplasia drug, which promotes endochondral bone growth, now covers children under five years of age with the rare genetic disease causing the most common form of dwarfism.

Amicus Therapeutics’ treatment for a rare muscle disorder called Pompe disease was approved by the U.S. health regulator, the drugmaker said on Thursday, ending its years-long efforts to launch the therapy.

The New England Journal of Medicine study suggests that the 2022 death of a Duchenne muscular dystrophy patient was due to an adverse effect of recombinant AAV used to deliver the gene therapy.

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.