Sarepta-Roche Gene Therapy Fails to Get EU Regulator Approval
The European Medicines Agency's drug advisory committee recommended against Elevidys for children with Duchenne muscular dystrophy, the agency said Friday. The medicine had been intended for children aged 3 to 7 years who are able to walk.
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Business Wire
4 hours ago
- Business Wire
Advicenne Receives Marketing Authorization and Reimbursement for Sibnayal® in Saudi Arabia
PARIS--(BUSINESS WIRE)--Regulatory News: Advicenne (Euronext Growth® - FR0013296746 - ALDVI), a pharmaceutical company specializing in the development and marketing of innovative treatments for people suffering from rare kidney diseases, obtains marketing authorization (MA) and reimbursement status for Sibnayal® (a fixed combination of potassium citrate and potassium bicarbonate) in the Kingdom of Saudi Arabia (KSA). Marketing authorization for Sibnayal® (ADV7103) in distal Renal Tubular Acidosis (dRTA) in KSA has been granted, based on the European registration dossier. Furthermore, Saudi authorities have agreed a reimbursement rate in line with the best European pricing. This achievment results of a close collaboration between Advicenne and its local partner, Taïba Healthcare, a leading distributor of pharmaceutical products in the Gulf region. Advicenne will act as the marketing authorization holder, while Taïba Healthcare will manage local marketing activities. The incidence of dRTA is higher in Gulf countries than in Europe or the United States, and prevalence in KSA could be estimated around 600 to 800 patients. Sibnayal® is already prescribed through early access programs in several Gulf countries outside Saudi Arabia. This milestone paves the way for registrations in GCC countries where the application is filed. Didier Laurens, CEO of Advicenne, declared: 'I am particularly proud of this important achievement and wish to congratulate both Advicenne and Taïba Healthcare teams, whose efforts were instrumental in informing and convincing the Saudi health authorities. The reimbursement obtained, comparable to the highest levels recorded in Europe, further attests to the significant therapeutic value of Sibnayal® in a region with one of the highest prevalence rates of dRTA worldwide.' About Advicenne Advicenne (Euronext: ALDVI) is a specialty pharmaceutical company founded in 2007, specializing in the development of innovative treatments in Nephrology. Its lead product Sibnayal® (ADV7103) has received its Marketing Approval for distal renal tubular acidosis in EU and GB. ADV7103 is currently in late-stage development in cystinuria in Europe and in dRTA and cystinuria in the US and in Canada. Headquartered in Paris, Advicenne, listed on the Euronext Paris stock exchange since 2017, has now been listed on Euronext Growth Paris since its transfer on March 30, 2022. For additional information, see: Disclaimer This press release contains certain forward-looking statements concerning Advicenne group and its business, including its prospects and product candidate development. Such forward-looking statements are based on assumptions that Advicenne considers to be reasonable. However, there can be no assurance that the estimates contained in such forward-looking statements will be verified, which estimates are subject to numerous risks including the risks set forth in the 2024 Universal Registration Document filed with the French financial market authority on April 29, 2025 (a copy of which is available on and to the development of economic conditions, financial markets and the markets in which Advicenne operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Advicenne or not currently considered material by Advicenne. The occurrence of all or part of such risks could cause actual results, financial conditions, performance, or achievements of Advicenne to be materially different from such forward-looking statements. Advicenne expressly declines any obligation to update such forward-looking statements.
Business Insider
11 hours ago
- Business Insider
Sarepta says eight-year-old death deemed unrelated to Elevidys
Sarepta (SRPT) said in a statement Friday night: 'Just before 6:00 p.m. ET today, the U.S. Food and Drug Administration issued a press release announcing an investigation into the death of an eight-year-old Duchenne muscular dystrophy patient who had received Elevidys gene therapy. The death of this patient was deemed unrelated to treatment with Elevidys…Sarepta reported this event to FDA on June 18, 2025, via the FDA's postmarketing electronic database, FAERS. At Sarepta, patient safety and well-being are always our top priority. We are committed to upholding the highest safety standards for all of our therapies, and do so in accordance with applicable law and commitment to full regulatory transparency. Elevidys is the only approved gene therapy for families and children devastated by Duchenne, a rare, progressive and ultimately fatal disease. We remain committed to working closely with the FDA to ensure that all decisions are grounded in science and the best interests of patients, considering the compelling need of these families to access disease-modifying therapy.' Elevate Your Investing Strategy:
Bloomberg
14 hours ago
- Bloomberg
Roche to Test Whether New Drug Can Prevent Alzheimer's Disease
Roche Holding AG plans to test whether an experimental medicine can prevent Alzheimer's disease symptoms in high-risk people, its latest investment in one of the most failure-prone areas of drugmaking. The new late-stage study will focus on people who are at risk of cognitive decline, Roche said in a statement late Sunday. The goal would be to slow down the emergence of symptoms, or prevent them entirely.
