This morning Amicus Therapeutics (FOLD 13.16, +2.90 +28.3%), a biotech firm specializing in therapies for rare and orphan diseases, announced plans to submit a new drug application to the US FDA for the oral precision medicine migalastat for Fibry disease in Q4. Shares trade to about 1 year 9 month highs on the update.
As a bit of background, Migalastat is an oral precision medicine intended to treat Fabry disease in patients who have amenable genetic mutations. Migalastat works by stabilizing the body's own dysfunctional enzyme, so it can clear the accumulated disease substrate in patients who have amenable mutations. An amenable mutation is one that is responsive to therapy with migalastat based on a proprietary in vitro assay. Amicus estimates that 35%-50% of Fabry patients globally may have amenable genetic mutations.
An estimated 3,000 people in the U.S. are currently diagnosed with Fabry disease. The U.S. represents the single largest geography for Amicus to positively impact the lives of people with Fabry who have amenable mutations.
Amicus is preparing the NDA submission under Subpart H, which provides for accelerated approval. The company intends to base its NDA on existing data, including reduction in disease-causing substrate (GL-3), as well as the totality of data from completed clinical studies. Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease and stroke. An additional Phase 3 study previously requested by the Agency to assess Gastrointestinal (GI) symptoms is no longer required prior to an NDA submission.
The European Commission (EC) has already granted full approval for migalastat under the trade name Galafold as a first line therapy for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease and who have an amenable mutation. Marketing applications have also been approved in several countries outside the EU, including Switzerland and Israel. Regulatory submissions are under review in additional countries including Japan, Canada and Australia.