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PTC Therapeutics (PTCT) Down on Negative CHMP Opinion on DMD Drug
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PTC Therapeutics (PTCT - Free Report) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Commission (EC) once again adopted a negative opinion on the annual renewal of the conditional marketing authorization of Translarna (ataluren) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD).
In response to the news, the company's shares plunged 11.8% on Friday. Translarna is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.
PTC Therapeutics plans to further request a re-examination of the negative opinion regarding Translarna in the EU. Meanwhile, the marketing authorization for Translarna will remain in effect until the re-examination procedure is completed and the EC announces its decision. Given the expected timeline for these procedures, PTCT anticipates that Translarna will remain available for boys and young men with nmDMD through the end of 2024, even if the negative opinion is ultimately upheld and ratified.
Please note that the CHMP had previously issued a negative opinion in January 2024 regarding the continued marketing approval of Translarna for the nmDMD indication. However, in May 2024, the EC decided against adopting the CHMP’s negative opinion and returned the same to the advisory committee for re-evaluation.
The EC had also instructed the CHMP to further consider the totality of evidence, including data from patient registries and real-world evidence, in the revised opinion.
Year to date, shares of PTC Therapeutics have gained 11% against the industry’s 7.8% decline.
Image Source: Zacks Investment Research
nmDMD is a genetic, rare and fatal muscle-wasting condition that typically affects males. It causes progressive muscle weakness from early childhood and leads to premature death in the mid-20s due to heart and respiratory failure.
In the United States, PTC Therapeutics plans to re-submit a new drug application (NDA) for Translarna for the treatment of nmDMD, which is based on the feedback from the FDA received earlier this year. The NDA resubmission is expected soon.
We remind the investors that the NDA pathway for potential Translarna approval in the United States has been nothing short of a rocky ride so far.
The NDA for Translarna was filed initially in 2017. However, the FDA issued a complete response letter in October 2017, stating that it was unable to approve the application in its current form.
After numerous discussions, meetings and FDA’s request for additional data, PTCT held a type C meeting with the FDA in the fourth quarter of 2023 to discuss the totality of Translarna data. Based on feedback from the regulatory body, PTCT plans to resubmit the NDA soon, supported by the results from Study 041 and its long-term, international drug registry study (STRIDE) for nmDMD patients receiving Translarna.
Zacks Rank and Stocks to Consider
PTC Therapeutics currently carries a Zacks Rank #3 (Hold).
In the past 30 days, the Zacks Consensus Estimate for ALX Oncology’s 2024 loss per share has remained constant at $2.89. During the same period, the consensus estimate for 2025 loss per share has remained constant at $2.73. Year to date, shares of ALXO have plunged 59.5%.
ALX Oncology beat estimates in two of the trailing four quarters and missed twice, delivering an average negative surprise of 8.83%.
In the past 30 days, the Zacks Consensus Estimate for Annovis’ 2024 loss per share has remained constant at $2.46. During the same period, the consensus estimate for 2025 loss per share has remained constant at $1.95. Year to date, shares of ANVS have plunged 69.2%.
ANVS beat estimates in three of the trailing four quarters and missed once, delivering an average negative surprise of 1.39%.
In the past 30 days, the Zacks Consensus Estimate for Compugen’s 2024 earnings per share has remained constant at 5 cents. The consensus estimate for 2025 loss per share is currently pegged at 11 cents. Year to date, shares of CGEN have lost 15.2%.
CGEN’s earnings beat estimates in three of the trailing four quarters and missed once, delivering an average surprise of 5.79%.
Image: Bigstock
PTC Therapeutics (PTCT) Down on Negative CHMP Opinion on DMD Drug
PTC Therapeutics (PTCT - Free Report) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Commission (EC) once again adopted a negative opinion on the annual renewal of the conditional marketing authorization of Translarna (ataluren) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD).
In response to the news, the company's shares plunged 11.8% on Friday. Translarna is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.
PTC Therapeutics plans to further request a re-examination of the negative opinion regarding Translarna in the EU. Meanwhile, the marketing authorization for Translarna will remain in effect until the re-examination procedure is completed and the EC announces its decision. Given the expected timeline for these procedures, PTCT anticipates that Translarna will remain available for boys and young men with nmDMD through the end of 2024, even if the negative opinion is ultimately upheld and ratified.
Please note that the CHMP had previously issued a negative opinion in January 2024 regarding the continued marketing approval of Translarna for the nmDMD indication. However, in May 2024, the EC decided against adopting the CHMP’s negative opinion and returned the same to the advisory committee for re-evaluation.
The EC had also instructed the CHMP to further consider the totality of evidence, including data from patient registries and real-world evidence, in the revised opinion.
Year to date, shares of PTC Therapeutics have gained 11% against the industry’s 7.8% decline.
Image Source: Zacks Investment Research
nmDMD is a genetic, rare and fatal muscle-wasting condition that typically affects males. It causes progressive muscle weakness from early childhood and leads to premature death in the mid-20s due to heart and respiratory failure.
In the United States, PTC Therapeutics plans to re-submit a new drug application (NDA) for Translarna for the treatment of nmDMD, which is based on the feedback from the FDA received earlier this year. The NDA resubmission is expected soon.
We remind the investors that the NDA pathway for potential Translarna approval in the United States has been nothing short of a rocky ride so far.
The NDA for Translarna was filed initially in 2017. However, the FDA issued a complete response letter in October 2017, stating that it was unable to approve the application in its current form.
After numerous discussions, meetings and FDA’s request for additional data, PTCT held a type C meeting with the FDA in the fourth quarter of 2023 to discuss the totality of Translarna data. Based on feedback from the regulatory body, PTCT plans to resubmit the NDA soon, supported by the results from Study 041 and its long-term, international drug registry study (STRIDE) for nmDMD patients receiving Translarna.
Zacks Rank and Stocks to Consider
PTC Therapeutics currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks from the drug/biotech industry are ALX Oncology Holdings (ALXO - Free Report) , Annovis Bio (ANVS - Free Report) and Compugen (CGEN - Free Report) , each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 30 days, the Zacks Consensus Estimate for ALX Oncology’s 2024 loss per share has remained constant at $2.89. During the same period, the consensus estimate for 2025 loss per share has remained constant at $2.73. Year to date, shares of ALXO have plunged 59.5%.
ALX Oncology beat estimates in two of the trailing four quarters and missed twice, delivering an average negative surprise of 8.83%.
In the past 30 days, the Zacks Consensus Estimate for Annovis’ 2024 loss per share has remained constant at $2.46. During the same period, the consensus estimate for 2025 loss per share has remained constant at $1.95. Year to date, shares of ANVS have plunged 69.2%.
ANVS beat estimates in three of the trailing four quarters and missed once, delivering an average negative surprise of 1.39%.
In the past 30 days, the Zacks Consensus Estimate for Compugen’s 2024 earnings per share has remained constant at 5 cents. The consensus estimate for 2025 loss per share is currently pegged at 11 cents. Year to date, shares of CGEN have lost 15.2%.
CGEN’s earnings beat estimates in three of the trailing four quarters and missed once, delivering an average surprise of 5.79%.