October 3, 2022

AstraZeneca’s rare disease unit Alexion will acquire gene editing specialist LogicBio Therapeutics in a small buyout deal, the companies announced Monday. Under the terms of the agreement, Alexion will acquire all of LogicBio’s outstanding shares for $2.07 each, representing a […]

October 3, 2022

Biotech company Zealand Pharma A/S, an innovator in peptide-based medicines, announced the positive topline results from its phase 3 trial of glepaglutide on Friday. A total of 106 patients with short-bowel syndrome (SBS) who also experienced intestinal failure and dependency […]

October 3, 2022

In draft guidance published on Friday, it emerged NICE does not recommend PTC Therapeutics’ Translarna (ataluren) for the treatment of Duchenne muscular dystrophy (DMD) caused by a nonsense mutation. The decision came following a re-evaluation of Translarna in order to […]

October 3, 2022

Amylyx Pharmaceuticals’ Relyvrio – a new treatment for amyotrophic lateral sclerosis (ALS) approved by the FDA on Thursday, after review of the data from its phase 2 trial – was the next day set at a list price of $158,000 […]

October 3, 2022

Pharma companies’ customer communication methods require reimagining as technology progresses. Kapoor says, now, pharma can learn from the on-demand nature of content distribution that Hollywood utilises and simultaneously prepare for future needs in content delivery. “One of our senior clients […]

September 30, 2022

The U.S. Food and Drug Administration on Thursday gave its approval to a new drug for ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig’s disease. But approval of the drug, called Relyvrio, is bound to stir new questions, with […]

September 30, 2022

Vanda Pharmaceuticals Inc. and OliPass Corp. have entered into a research and development collaboration to jointly develop a set of antisense oligonucleotide (ASO) molecules based on OliPass’ modified peptide nucleic acids. The partnership leverages the respective strengths of Vanda and […]

September 30, 2022

Roctavian’s road back to the FDA has been long, as BioMarin has had to gather additional data in support of its therapy. Delays and new safety concerns further hampered the company’s resubmission plans. The treatment is for people with severe […]

September 30, 2022

The accelerated approval program was implemented as the U.S. grappled with the HIV crisis and was searching for a way to get medicines for the deadly infection to market more quickly. But its use has skyrocketed in recent years, with […]

September 29, 2022

Sarepta Therapeutics has asked the Food and Drug Administration to approve its gene therapy for Duchenne muscular dystrophy under accelerated review, the company said Thursday. The biotechnology company’s application for SRP-9001, also known as delandistrogene moxeparvovec, is primarily based on […]