The PTC Therapeutics drug Translarna failed its confirmatory study in Duchenne muscular dystrophy. Analysts say the European Commission’s decision to not adopt the Committee on Medicinal Products for Human Use’s negative opinion on the drug is unusual, if not unprecedented.
PTC Therapeutics plans to talk to regulators about regulatory submissions for sepiapterin after the drug met the main goal of a Phase 3 study. The small molecule is a potential treatment for phenylketonuria, a rare enzyme deficiency.
Munck Wilson Mandala Partner Greg Howison shared his perspective on some of the legal ramifications around AI, IP, connected devices and the data they generate, in response to emailed questions.
Roche drug Evrysdi, one of three FDA-approved treatments for spinal muscular atrophy, is now approved for treating those younger than 2 months of age. The regulatory decision puts the oral drug on equal footing with SMA drugs from Novartis and Biogen.
Analysts noted that Evrysdi would be priced significantly lower than Biogen's Spinraza or Novartis' Zolgensma. While this will provide a market advantage, it would also yield lower per-patient revenues.
The company hopes that PTC299's dual mechanism of action can both inhibit replication of the SARS-CoV-2 while alleviating the inflammation associated with severe Covid-19.
An analyst wrote that while the delay for risdiplam - prompted by Roche's submission of additional data - is unfortunate, the timing of the pivotal trials and the patient population guarantee a broad label for the drug, making the company's decision prudent.
Gabby Everett, the site director for BioLabs Pegasus Park, offered a tour of the space and shared some examples of why early-stage life science companies should choose North Texas.
As a whole host of new Duchenne Muscular Dystrophy therapies mature down the regulatory pathway, the global market for the degenerative disease is expected to expand at an incredible rate – from $8.2 million in 2014 to $990 million in 2019, or so says market research firm GlobalData. The present market’s got only paltry offerings to treat this […]
(Reuters) - PTC Therapeutics Inc said a committee of the European Medicines Agency did not recommend conditional approval of its drug for the treatment of a rare muscular disorder, sending its shares down as much as 28 percent in premarket trading. The...
A biopharmaceutical company with a cystic fibrosis drug in late-stage development has raised $30 million, according to a company statement. It was about $10 million less than the original funding target listed in a form D document with the U.S. Securities and Exchange Commission last month. The target of PTC Therapeutics‘ small molecule drug Ataluren […]
A drug developer that recently reported positive phase 3 results for its cystic fibrosis treatment for a severe form of the genetic disorder is raising $40 million in equity, according to a Form D filing with the U.S. Securities and Exchange Commission. So far it has raised more than half that amount, according to the […]
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
PTC Therapeutics has received a $5 million Seeding Drug Discovery award from UK charity Wellcome Trust to support the development of small-molecule drugs to battle life-threatening infections caused by multi-drug-resistant, Gram-negative bacteria. The South Plainfield, New Jersey-based drug development company’s anti-infective program is “currently in lead optimization and advancing toward identifying a development candidate,” according […]
PTC Therapeutics has agreed to a licensing deal with Swiss pharmaceutical firm Roche for its spinal muscular atrophy program that could be worth as much as $490 million. Under the terms of the agreement, Roche gains an exclusive worldwide license to PTC’s SMA program, which includes three compounds currently in preclinical development, as well as […]