PARIS – The European Medicines Agency has issued a marketing authorisation for OZAWADE™ (pitolisant), indicated in the treatment of excessive daytime sleepiness (EDS) associated with obstructive sleep apnoea in adults. OZAWADE™ is indicated to improve wakefulness and reduce EDS in adult patients suffering from obstructive sleep apnoea (OSA) whose EDS...
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Paris — The European Medicines Agency has just granted an extension of the indication for the marketing authorisation for WAKIX® (pitolisant), which is now indicated for the treatment of narcolepsy in children from the age of 6 years, with or without cataplexy. Narcolepsy is a rare disease, which originates in...
MIAMI – Biorasi, a leading global full-service contract research organization (CRO), has announced the appointment of Jimmy El Hokayem, Ph.D., head of the company’s Neurology and Regenerative Medicine Center of Excellence to the Scientific Advisory Board (SAB) for the Foundation for Angelman Syndrome Therapeutics (FAST). In this role, Dr. El Hokayem...
Washington, DC – In 2023, cell and gene therapy saw an unprecedented surge with seven FDA approvals, and this year, an even greater number of these treatments could reach the market. So far in 2024, the regulator has given the green light to three new CGTs, and at least seven...
Washington, DC – The March approval of ITF Therapeutics’ Duvyzat marked the third new FDA-approved treatment in 10 months for Duchenne muscular dystrophy (DMD), a genetic disorder that causes progressive muscle weakness and degeneration. In June 2023, the regulator greenlit Sarepta’s Elevidys as the first-ever gene therapy for DMD. That...
Basel, Switzerland – Novartis on Saturday posted two promising Phase III readouts in rare kidney diseases, setting the pharma up for two potential approvals in the space. The company presented these data at the European Renal Association Congress over the weekend. Results from the Phase III ALIGN study showed that...
Washinton, DC – On March 15, the FDA’s Oncologic Drugs Advisory Committee will meet to discuss two CAR T cell therapies for multiple myeloma—Abecma, from Bristol Myers Squibb and 2seventy bio, and Carvykti, from Johnson & Johnson and Legend Biotech. Both treatments are currently approved for the treatment of adults...
Washington, DC – In February 2024, Larimar Therapeutics released positive Phase II data for its injectable subcutaneous investigational agent nomlabofusp in treating Friedreich’s ataxia, a rare disease that causes neuromuscular degeneration. The data indicate that Larimar could go head-to-head in the market with Biogen’s Skyclarys, the only disease-specific therapy for Friedreich’s...
Washington, DC – As the oncology space moves away from single oncogene-targeted treatments, immunotherapies that aim to prevent immune fatigue and enhance immune cell engagement are emerging as promising therapeutic trends, experts told BioSpace. But these therapies are not always intended to be deployed on their own. Increasingly, scientists and...
Dallas, Texas – Taysha Gene Therapies touted positive early-stage data last month for its gene therapy to treat Rett syndrome, which has become the company’s main focus after cutting other programs and laying off 35% of its staff. If successful, the candidate for Rett syndrome has the capability to put...