Ultragenyx (RARE) Falls 26% as New Treatment Fails to Get FDA Green Light

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the stocks drowned heavily. Are you holding any?

Ultragenyx fell by 26.05 percent week-on-week as investor sentiment was largely dragged down by the Food and Drug Administration’s (FDA) rejection of its biologics license application (BLA) for its UX111 (ABO-102) treatment.

In a regulatory filing, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said that the FDA requested additional information and improvements in relation to certain chemistry, manufacturing, and controls (CMC) aspects.

It noted that the observations were not related to product quality concerns product, adding that many of the issues have already been addressed.

UX111 was developed to treat a common type of Sanfilippo syndrome, a group of genetic conditions that begin in early childhood and causes severe brain damage and early death.

A research team in a laboratory peering through microscopes at a biologic product.

The treatment involves managing symptoms, as there are currently no approved disease-modifying medicines.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said it plans to resubmit updated clinical data from current patients after resolving the FDA’s concerns. A new review could take another six months upon resubmission of a revised BLA application.

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Disclosure: None. This article is originally published at Insider Monkey.