📣 PTC Therapeutics Informa:

Actualización para la Comunidad Duchenne en EE. UU. sobre la revisión de la solicitud de registro de nuevo fármaco de Ataluren (Translarna™)

#TheAkariFoundation #enfermedadesraras #DistrofiaMuscularDeDuchenne #duchenneenespañol #PTCTherapeutics #Ataluren #Translarna

PTC pulls file for Duchenne therapy on FDA feedback PTC Therapeutics has abandoned its latest attempt to secure FDA approval for Duchenne muscular dystrophy treatment Translarna.

#PTCTherapeutics has abandoned its latest attempt to secure #FDA approval for #Duchenne muscular dystrophy (DMD) treatment #Translarna, after the FDA said it was unlikely to approve the drug based on the submitted data.

US FDA declines to approve PTC Therapeutics’ oral drug for rare genetic disorder By Puyaan Singh and Kamal Choudhury (Reuters) -The U.S. Food and Drug Administration has denied approval for PTC Therapeutics (NASDAQ:PTCT)’ oral medication for a rare genetic disorder with limited treatment options, the company said on Tuesday. In its so-called Complete Response Letter, the FDA said the data did not provide substantial efficacy evidence for the drug, vatiquinone, in treating Friedreich’s ataxia and that a separate study was needed before resubmitting the application. At least three analysts said the regulator’s rejection was not surprising and that expectations had been low heading into the decision. PTC (NASDAQ:PTC)’s shares reversed premarket losses to rise more than 8% in morning trading. The FDA approved Sephience - PTC’s oral therapy for a rare metabolic disorder called phenylketonuria - last month, potentially countering revenue declines due to regulatory scrutiny and competition from cheaper rivals for its top-selling muscular disorder therapies, Translarna and Emflaza. "We see today’s CRL as a clearing event since investors can now turn their focus to the Sephience launch," J.P.Morgan analysts said in a client note. The company said it planned to meet with FDA officials to discuss potential next steps. "We are of course disappointed by the FDA’s decision to not approve vatiquinone," CEO Matthew Klein said, adding that the data till date demonstrated that the drug could be safe and effective for children and adults living with the disorder. Friedreich’s ataxia is a rare genetic disorder that causes progressive damage to the nervous system, often leading to difficulty in walking, speech problems and heart complications. The disease tends to develop in children and teenagers and gradually worsens over time. Currently, Biogen (NASDAQ:BIIB)’s Skyclarys remains the only FDA-approved treatment for Friedreich’s ataxia. Don't miss out on the next big opportunity! Stay ahead of the curve with ProPicks AI – 6 model portfolios fueled by AI stock picks with a stellar performance this year... In 2024 alone, ProPicks AI identified 2 stocks that surged over 150%, 4 additional stocks that leaped over 30%, and 3 more that climbed over 25%. That's an impressive track record. With portfolios tailored for Dow stocks, S&P stocks, Tech Stocks, and Mid Cap stocks, you can explore various wealth-building strategies. So if BIIB is on your watchlist, it could be very wise to know whether or not it made the ProPicks AI lists.

Click Subscribe #FDA #PTCTherapeutics #DrugApproval #GeneticDisorder #RareDiseases

Actualización para la Comunidad Duchenne en EE. UU. sobre la revisión de la solicitud de registro de un fármaco (NDA, por sus siglas en inglés) de Ataluren (Translarna).

#TheAkariFoundation #enfermedadesraras #DistrofiaMuscularDeDuchenne #duchenneenespañol #PTCTherapeutics #Ataluren

Germany will be first launch for PTC's phenylketonuria drug PTC Therapeutics' treatment for the rare disease PKU, Sephience, will launch in its first market next month after getting EU approval.

The #EuropeanCommission has approved #PTCTherapeutics' treatment for the rare disease phenylketonuria (#PKU), #Sephience, setting up a market debut in Germany next month.

pharmaphorum.com/news/germany...

uniQure Announces Appointment of Kylie O’Keefe as Chief Customer and Strategy Officer ~ Proven biotech executive to lead commercialization of AMT-130 in Huntington’s disease ~ LEXINGTON, Mass. and AMSTERDAM, June 11, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene ...

#uniQure hires away Kylie O’Keefe from #Huntington's competitor #PTCTherapeutics, planning for AMT-130 launch.

Guess she has more confidence in uniQure's ddRNAi over PTC's small molecule reaching market soon.

So do I.

finance.yahoo.com/news/uniqure...

The external comparator arm will be from the ENROLL-HD study. Pretty sure #uniQure would not have agreed on that if they felt it was losing proposition. Based on #PTCTherapeutics data, cUHDRS declines in ENROLL-HD substantial.

Este evento fue un espacio de comunidad, aprendizaje y esperanza. Vernos, escucharnos y apoyarnos nos recuerda que no estamos solos en este camino.🤝💙

#SareptaTherapeutics #NSPharma #EdgewiseTherapeutics #PTCTherapeutics #CatalystPharmaceuticals #ITFTherapeutics #AvidityBiosciences

So yeah, #Novartis had seen #PTCTherapeutics 12-month PIVOT-HD data.

#PTCTherapeutics claims win on 'primary endpoint', huntingtin-lowering IN BLOOD! But look at more relevant CSF measurement.

#UniQure

Wishful thinking as this is an oral drug.

But yes, if #PTCTherapeutics can confirm last year's data in Q2, then it would be a positive for #WaveLifeSciences. #UniQure #Huntington'sDisease

Kebilidi, AADC deficiency gene therapy, now approved by FDA The U.S. Food and Drug Administration has approved PTC Therapeutics' one-time gene therapy, called Kebilidi, for people with AADC deficiency.

Gene therapy Kebilidi now approved in the US to treat those with AADC deficiency. aadcnews.com/news/kebilid...

#ptctherapeutics #kebilidi #aadc #rarediseases