Parents Push One Rare-Disease Drug Through FDA, Not Its Successor
The agency’s rejection of a new therapy for treating a deadly condition may signal a tougher FDA, and more risks for makers of rare-disease drugs......»»
ChemoCentryx"s stock dives after critique from the FDA about experimental treatment for rare autoimmune disease
Shares of ChemoCentryx Inc. plunged 44.8% in trading on Tuesday after the Food and Drug Administration questioned the rat.....»»
Akouos receives orphan drug and rare pediatric disease designations for AK-OTOF
See the rest of the story here. Theflyonthewall.com provides the latest financial news as it breaks. Known as a leader in market intelligence, The Fl.....»»
Ionis shares tumble 19% after Roche halts Phase 3 study for rare-disease drug
Shares of Ionis Pharmaceuticals Inc. were down 19.6% in premarket trading on Tuesday, the day after the company said Roche Holding AG decided to discontinue.....»»
Ionis shares tumble19% after Roche halts Phase 3 study for rare-disease drug
Shares of Ionis Pharmaceuticals Inc. were down 19.6% in premarket trading on Tuesday, the day after the company said Roche Holding AG decided to discontinue.....»»
Tackling deadly newborn disease, BridgeBio affiliate wins FDA drug approval
The BridgeBio model is designed to move drugs tackling rare diseases with a well-defined genetic basis quickly and efficiently to approval......»»
SVB Leerink, J.P. Morgan downgrade Amicus stock after Phase 3 data for rare disease drug comes out
Shares of Amicus Therapeutics Inc. were down 23.1% in premarket trading on Friday, the day after the company said an experimental rare disease d.....»»
Taysha gets both rare pediatric disease and orphan drug designations for TSHA-10
See the rest of the story here. Theflyonthewall.com provides the latest financial news as it breaks. Known as a leader in market intelligence, The Fl.....»»
Prevail receives Orphan Drug, Rare Pediatric Disease designations for PR001
See the rest of the story here. Theflyonthewall.com provides the latest financial news as it breaks. Known as a leader in market intelligence, The Fl.....»»
Families of rare-disease patients take on drug development, but face uphill battle
Frustrated with the slow pace of drug discovery and desperate to help their loved ones, untrained family members take the work into their own hands by starting biopharma companies. But few such companies have been successful......»»
The Daily Biotech Pulse: Osmotica Soars On Insider Buys, Ultragenyx"s Partnered Rare Disease Drug Moves Into Clinics
Here's a roundup of top developments in the biotech space over the last 24 hours. Scaling The Peaks (Biotech Stocks Hitting 52-week highs on Jan. 15) 0 read more.....»»
A 4-year-old girl in Iowa went blind after the flu caused a rare disease to impair her brain function
David Goldman/Getty Jade DeLucia, a 4-year-old in Iowa, was not vaccinated this flu season and wound up in intensive care. On January 1, her parents discovered that the flu had affected the p.....»»
Sarepta’s surprise FDA approval for rare disease drug excites investors
The Food and Drug Administration’s out-of-the-blue approval of Sarepta Therapeutics’ second Duchenne muscular dystrophy drug sent the company’s stock soaring......»»
In an about face, FDA approves Sarepta"s 2nd Duchenne drug
The FDA unexpectedly reversed course Thursday, approving the second treatment developed by Sarepta Therapeutics for the rare disease Duchenne muscular dystrophy just a few months after rejecting the drug. Sarepta (Nasdaq: SRPT) announced Thursday even.....»»
The Ratings Game: Drug maker ChemoCentryx shares rocket on blockbuster potential in rare autoimmune disease
A set of conclusive findings from a late-stage clinical trial testing ChemoCentryx Inc.’s experimental vasculitis treatment surprised company executives and investors, who now say they are bullish on the sales and pipeline potential of avacopan......»»
Celgene"s rare disease drug gets FDA approval
Shares of Celgene Corp. [s:celg] rose 0.34% in afternoon trading after the U.S. Food and Drug Administration approved Reblozyl. The drug, which treats anemia in patients with beta thalassemia, a rare blood disorder, was developed jointl.....»»
Drug to treat a rare disease costs millions
Dawn Patterson keeps a multimillion-dollar drug in the fridge, next to a bottle of root beer and a jar of salsa......»»
Arrowhead Looks To Get Rare Liver Disease Drug Approved With Use Of Accelerated Approval
Arrowhead Looks To Get Rare Liver Disease Drug Approved With Use Of Accelerated Approval.....»»
Undeterred by Vertex"s plans, biotech Dyne enters Duchenne drug fray
The field of biotechs developing treatments for Duchenne muscular dystrophy became more crowded this week, with biotech startup Dyne setting its sights on the muscle-wasting rare disease......»»
The costliest drug on the planet will treat infants with rare disease. The market fight focused on cost and safety is just getting started.
Infants with spinal muscular atrophy may soon have two treatment options......»»
The costliest drug on the planet will treat infants with rare disease.
Infants with spinal muscular atrophy may soon have two treatment options......»»