Ultragenyx Pharmaceutical, Inc. Stock News

Ultragenyx Pharmaceuticals won approval for its newest gene therapy on Thursday, helping shares partially reverse a catastrophic dive.

FAYUVI is a highly anticipated, first-ever treatment option with the potential to stop or slow the devastating, irreversible neurologic progression and loss of function associated with Sanfilippo syndrome Type A

The U.S ​Food ‌and Drug ​Administration ​said on Thursday ⁠it ​has ​approved Ultragenyx's gene ​therapy ​for the treatment ‌of ⁠a rare, fatal ​disease.

Ultragenyx stock crashed Thursday, losing almost half its value, after its experimental Angelman treatment failed in final-phase testing.

Ultragenyx Pharmaceuticals looks to cut costs following the trial failure, while attention remains squared on its burgeoning commercial business.

Shares of Ultragenyx Pharmaceutical plunged more than 40% in premarket on Thursday after its neurodevelopmental disorder treatment failed a closely watched ​late-stage trial, dealing a major blow to one of ‌its biggest growth bets.

Ultragenyx Pharmaceutical said on ‌Wednesday its experimental drug for a rare genetic neurodevelopmental disorder did not ​meet the main goal in ​a late-stage trial, sending its shares ⁠down 40% in aftermarket trading.

Phase 3 Aspire did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI)