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PTC Therapeutics (PTCT) Up on Positive Regulatory Updates

PTC Therapeutics, Inc. PTCT announced that it has submitted a biologics license application (BLA) for gene therapy Upstaza (eladocagene exuparvovec) to the FDA.

The BLA is seeking approval of the therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency, a fatal, rare genetic disorder that typically causes severe disability in babies.

Upstaza has received marketing authorization in Europe, Great Britain and Israel. It is a one-time gene replacement therapy indicated for the treatment of patients aged 18 months and older with a clinical, molecular and genetically confirmed diagnosis of AADC deficiency with a severe phenotype.

Concurrently, PTC Therapeutics announced plans to re-submit a new drug application (NDA) for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD), based on recent feedback from the FDA.

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The NDA resubmission is targeted by the middle of 2024. 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.

Meanwhile, the company also announced that the marketing authorization application for sepiapterin for the treatment of phenylketonuria remains on schedule for submission to the European Medicines Agency EMA by the end of this month.

Shares of PTCT rose 4.89% on these positive regulatory updates.

The company’s shares lost 38.8% in the past year compared with the industry’s 3.9% decline.

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Translarna has faced a few roadblocks in its NDA pathway.  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 this discussion, the FDA suggested PTCT to request a pre-submission type C meeting to discuss the specific contents of an NDA resubmission based on the results from Study 041 and its international drug registry study for nmDMD patients receiving Translarna.

In January 2024, the Committee for Medicinal Products for Human Use of the European Medicines Agency issued a negative opinion for the conditional marketing authorization of Translarna for the treatment of nmDMD, following a re-examination procedure.

Meanwhile, PTCT’s other drug, Emflaza, is approved in the United States for Duchenne muscular dystrophy.

PTCT has also collaborated with Swiss pharma giant Roche RHHBY for the development of Evrysdi, a treatment for spinal muscular atrophy (SMA). The drug was approved by the FDA for the treatment of SMA in adults and children of all ages. It also received approval from the European Commission.

Roche acquired an exclusive worldwide license to the company’s SMA program in 2011 in return for milestone and royalty payments.

Zacks Rank & Stocks to Consider

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

Some better-ranked stocks in the healthcare sector are ADMA Biologics, Inc. ADMA and ANI Pharmaceuticals, Inc. ANIP, both sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for ADMA Biologics’ 2024 earnings per share (EPS) have improved from 22 cents to 30 cents. In the past year, shares of ADMA have rallied 99.7%.

ADMA Biologics’ earnings beat estimates in three of the trailing four quarters and met the same once, delivering an average surprise of 85.00%.

In the past 60 days, estimates for ANI Pharmaceuticals’ 2024 EPS have improved from $4.06 to $4.40. In the past year, shares of ANIP have surged 70.3%.

ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 109.06%.

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Roche Holding AG (RHHBY) : Free Stock Analysis Report

PTC Therapeutics, Inc. (PTCT) : Free Stock Analysis Report

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