Back to top

Image: Bigstock

PTC Therapeutics (PTCT) Down on Negative CHMP Opinion for DMD Drug

Read MoreHide Full Article

PTC Therapeutics (PTCT - Free Report) announced that it received a negative opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for converting conditional marketing of Translarna (ataluren) to full marketing authorization. Shares of the company were down 30% on Sep 18, following the news.

Furthermore, the negative opinion also applies to the renewal of the existing conditional authorization of Translarna, which had received conditional marketing authorization in Europe in 2014 to treat nonsense mutation Duchenne muscular dystrophy (nmDMD). This authorization was subsequently renewed in 2017.

In response to the negative opinion, PTCT plans to request a formal re-examination procedure of the regulatory application per the EMA guidelines. The re-examination process is expected to occur in January 2024. Translarna will continue to remain on the market and available to patients with nmDMD until the re-examination process is completed.

Shares of PTC Therapeutics have risen 20.8% year to date against the industry’s 14.0% decline.

Zacks Investment Research
Image Source: Zacks Investment Research

The CHMP’s decision was based on data from three placebo-controlled studies (Study-007, Study-041 and Study-020) of the Translarna therapy. In addition to the clinical study data, the CHMP considered additional supportive evidence of the drug's efficacy. This included the results of robust meta-analyses, which are comprehensive statistical analyses that showed statistically significant benefits.

The analysis of the real-world STRIDE registry data was also considered by CHMP. The data from this registry indicated that long-term Translarna therapy could delay the time it takes for patients to lose their ability to walk.

In the re-examination process, PTCT intends to focus on the clear and consistent evidence of benefit recorded in the overall population of 700 boys included in all three studies. It will also address concerns raised regarding the robustness of data from a STRIDE registry, which includes 300 boys with an average treatment duration of more than 5.5 years, supporting the benefits of long-term treatment.

Zacks Rank & Stocks to Consider

PTC Therapeutics currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the same industry are Anika Therapeutics (ANIK - Free Report) , Annovis Bio (ANVS - Free Report) and ANI Pharmaceuticals (ANIP - 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 90 days, the Zacks Consensus Estimate forAnika Therapeutics has narrowed from a loss of $1.41 per share to a loss of $1.24 for 2023. The bottom-line estimate has widened from a loss of 79 cents to a loss of 82 cents for 2024 during the same time frame. Shares of the company have lost 41.0% year to date.

ANIK’s earnings beat estimates in one of the trailing four quarters and missed the mark in the remaining three, delivering an average negative surprise of 32.12%.

In the past 90 days, the Zacks Consensus Estimate for Annovis Bio has narrowed from a loss of $4.89 per share to a loss of $4.38 for 2023. The bottom-line estimate has narrowed from a loss of $3.18 to a loss of $2.77 for 2024 during the same time frame. Shares of the company have lost 21.0% year to date.

ANVS’ earnings beat estimates in three of the trailing four quarters and missed the mark in one, delivering an average surprise of 13.40%.

In the past 90 days, the Zacks Consensus Estimate for ANI Pharmaceuticals’ earnings has gone up from $3.31 per share to $4.02 for 2023. The bottom-line estimate has increased from $4.32 to $4.59 for 2024 during the same time frame. Shares of the company have rallied 55.1% year to date.

ANIP’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 91.56%. 


Zacks' 7 Best Strong Buy Stocks (New Research Report)


Valued at $99, click below to receive our just-released report
predicting the 7 stocks that will soar highest in the coming month.


Click Here, It's Really Free

Published in