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NEW YORK (AP) — In a story Jan. 3 about Repligen Corp. partnering with Pfizer Inc. to develop a drug to treat spinal muscular atrophy, The Associated Press erroneously reported the amount of money that Repligen could receive from Pfizer. Repligen is entitled to receive up to $70 million, including a $5 million upfront payment and up to $65 million in potential future milestone payments.
A corrected version of the story is below:
Repligen forms partnership on spinal muscle drug
Repligen will work with Pfizer to develop drug to treat for Spinal Muscular Atrophy
NEW YORK (AP) — Repligen Corp. said Thursday that it will partner with Pfizer Inc. to develop a drug to treat spinal muscular atrophy, a rare genetic disorder that is one of the leading causes of infant deaths.
The Waltham, Mass., company said it will get $5 million upfront from Pfizer, and could receive an additional $65 million in future milestone payments. Repligen would also receive royalty payments on any approved products. The deal gives Pfizer one drug in clinical testing and other compounds and technologies.
The drug, designated RG309, is being tested on healthy people in an early-stage clinical trial. Repligen said it will continue that study and complete safety testing of the drug on some groups of volunteers by the end of March. Pfizer will then take over developing the product.
Spinal muscular atrophy causes progressive damage to motor neurons. Symptoms typically appear before age 2, and the disease often causes early death because of respiratory problems. Repligen says RG309 is the first drug being developed specifically to treat spinal muscular atrophy and it has received fast-track status from the Food and Drug Administration.
Fast-track status is aimed at speeding up the approval of drugs that treat serious or life-threatening diseases for which there are few other therapies. The drug also has received orphan drug designation in the U.S. and the European Union, another development incentive which means that if approved, RG309 would be free of product competition for up to seven years in the U.S. and 10 years in Europe.