US FDA approves Ryplazim: the first drug to treat plasminogen deficiency

Liminal BioSciences recently announced that the U.

While approving Ryplazim, the FDA also issued a rare pediatric disease priority review voucher (PRV) to Liminal BioSciences to reward the company for its outstanding contribution to the development of new drugs for rare diseases

Bruce Pritchard, CEO of Liminal BioSciences, said: "This approval is an important milestone for Liminal, patients, caregivers and doctors

Plasminogen deficiency is a rare multi-system and genetic disease that has a profound impact on the health and quality of life of patients

Plasminogen is a naturally occurring protein that is synthesized by the liver and circulates in the blood

The active ingredient of Ryplazim is plasminogen, which is extracted and purified from human plasma

The efficacy of Ryplazim in pediatric and adult patients with plasminogen deficiency type 1 was confirmed in a single-arm, open-label clinical trial

Efficacy is based on the total clinical success rate at 48 weeks, defined as 50% of patients with visible or other measurable invisible lesions, and at least a 50% improvement in the number/size or functional impact of lesions from baseline

The approval of Ryplazim for marketing marks an important turning point, providing patients with type 1 plasminogen deficiency with the first and much-needed therapy, and providing patients and families with a new option to help control the symptoms of the disease

Original source: Liminal BioSciences Announces FDA Approval for its Biologics License application for Ryplazim® (plasminogen, human-tvmh)