This section highlights FDA-related milestones and regulatory updates for drugs developed by PTC Therapeutics (PTCT).
Over the past two years, PTC Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
PTC518, Sepiapterin, Translarna™, Upstaza, Utreloxastat, and Vatiquinone. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
PTC518 - FDA Regulatory Timeline and Events
PTC518 is a drug developed by PTC Therapeutics for the following indication: Huntington's Disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- PTC518
- Announced Date:
- May 5, 2025
- Indication:
- Huntington's Disease
Announcement
PTC Therapeutics, Inc. announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease (HD) patients.
AI Summary
PTC Therapeutics recently announced promising results from its Phase 2 PIVOT-HD study evaluating PTC518 (votoplam) in Stage 2 and Stage 3 Huntington’s disease patients. The study met its primary endpoint by showing a significant, dose-dependent lowering of blood Huntingtin (HTT) protein levels at week 12. In Stage 2 patients, favorable trends were noted on clinical scales at Month 12, with reductions of approximately 23% at the 5mg dose and up to 39% at the 10mg dose.
At 24 months, the study revealed dose-dependent clinical benefits compared to a matched natural history cohort, along with a reduction in neurofilament light chain (NfL) levels. PTC518 maintained a favorable safety and tolerability profile with no treatment-related NfL spikes, suggesting its potential as a disease-modifying therapy for Huntington’s disease.
Read Announcement- Drug:
- PTC518
- Announced Date:
- September 26, 2024
- Indication:
- Huntington's Disease
Announcement
PTC Therapeutics announced today that the FDA has granted Fast Track designation to the PTC518 program for the treatment of Huntington's disease. Fast Track designation is awarded to promising therapies for diseases of high unmet need.
AI Summary
PTC Therapeutics announced that the FDA has granted Fast Track designation to its experimental drug, PTC518, for the treatment of Huntington’s disease. This designation is given to promising therapies that address serious conditions with high unmet needs, allowing for earlier and more frequent engagements with the FDA while expediting the review process. The Fast Track status underscores the potential of PTC518 to modify the disease course by reducing the production of the mutated Huntingtin protein, which is central to the disease’s progression.
Matthew B. Klein, M.D., CEO of PTC Therapeutics, emphasized the promising outlook for PTC518, noting that the program was discovered using the company’s innovative splicing platform. The collaborative efforts with the FDA aim to advance PTC518’s development as efficiently as possible, offering new hope to patients with Huntington’s disease.
Read Announcement- Drug:
- PTC518
- Announced Date:
- June 20, 2024
- Indication:
- Huntington's Disease
Announcement
PTC Therapeutics, Inc shared interim results from the Phase 2 PIVOT-HD study of PTC518 in Huntington's disease (HD) patients. At Month 12, PTC518 treatment resulted in dose-dependent lowering of mutant huntingtin (mHTT) protein in the blood and cerebrospinal fluid (CSF) in the interim cohort of patients.
AI Summary
PTC Therapeutics recently shared promising interim results from the Phase 2 PIVOT-HD study of its investigational drug PTC518 for Huntington’s disease. At Month 12, the treatment showed a dose-dependent decrease in mutant huntingtin (mHTT) protein levels in both blood and cerebrospinal fluid. Specifically, for the 5mg dose, mHTT levels dropped by about 22% in blood and 21% in the CSF, while the 10mg dose achieved reductions of 43% in both compartments.
Additionally, early clinical assessments indicated a slowing of motor symptom progression compared to placebo, along with a favorable tolerability profile after one year of treatment. These findings support further development of PTC518 as a potential disease-modifying therapy for patients with Huntington’s disease, offering hope for a treatment that targets the underlying protein abnormality and may slow disease progression.
Read Announcement
Sepiapterin - FDA Regulatory Timeline and Events
Sepiapterin is a drug developed by PTC Therapeutics for the following indication: To treat the broad range of PKU patients.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Sepiapterin
- Announced Date:
- April 25, 2025
- Indication:
- To treat the broad range of PKU patients.
Announcement
PTC Therapeutics, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU).
AI Summary
PTC Therapeutics, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a positive opinion on the marketing authorization application for Sephience™ (sepiapterin). This decision supports the use of Sephience for the treatment of phenylketonuria (PKU) in both children and adults, offering a broad label that includes patients of all ages and disease severities. The drug works using a dual mechanism that not only lowers phenylalanine levels but also has the potential to allow for more dietary flexibility even in severe cases of PKU. PTC is making significant launch preparations in Europe, with key plans already in place for markets like Germany. The European Commission is expected to formally adopt this positive opinion soon, which will enable access to Sephience across all 27 EU member states, as well as in Iceland, Norway, and Liechtenstein.
Read Announcement- Drug:
- Sepiapterin
- Announced Date:
- March 20, 2025
- Indication:
- To treat the broad range of PKU patients.
Announcement
PTC Therapeutics, Inc. today shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting.
AI Summary
PTC Therapeutics, Inc. presented new data from its Phase 3 APHENITY trial and follow-up open-label extension study at the 2025 American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting. The results showed that more than 97% of the study’s PKU patients were able to increase their dietary phenylalanine (Phe) intake, with a mean increase of 126% in protein consumption. The trial also indicated that 66% of the participants met or exceeded the age-adjusted recommended daily protein allowance without compromising blood Phe levels.
Furthermore, genetic variant analysis revealed that over 70% of the subjects had a classical PKU phenotype, including those with non-BH4 responsive mutations. These findings suggest that sepiapterin treatment can help a broad spectrum of PKU patients by improving Phe tolerance and supporting better dietary management.
Read Announcement- Drug:
- Sepiapterin
- Announced Date:
- October 1, 2024
- Indication:
- To treat the broad range of PKU patients.
Announcement
PTC Therapeutics, Inc. announced the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for the treatment of pediatric and adult patients living with phenylketonuria (PKU).
AI Summary
PTC Therapeutics, Inc. recently reached an important milestone when the FDA accepted for filing its New Drug Application (NDA) for sepiapterin, a novel treatment aimed at both pediatric and adult patients with phenylketonuria (PKU). This regulatory step is crucial as it marks the transition to the review phase, where the agency will assess the safety and efficacy of sepiapterin for managing PKU. The acceptance for filing underlines the potential of sepiapterin as a promising therapeutic option for a condition that poses significant challenges to those affected.
The filing demonstrates PTC Therapeutics’ commitment to addressing unmet medical needs in rare disorders. By advancing the development of sepiapterin, the company hopes to provide a transformative treatment that improves the quality of life for those living with PKU. Future updates will detail the progress of the regulatory review and potential clinical benefits for patients.
Read Announcement- Drug:
- Sepiapterin
- Announced Date:
- October 1, 2024
- Indication:
- To treat the broad range of PKU patients.
Announcement
PTC Therapeutics, Inc announced that A Prescription Drug User Fee Act (PDUFA) target action date is expected to be provided in the Day 74 Letter.
AI Summary
PTC Therapeutics recently announced that the FDA is expected to provide a Prescription Drug User Fee Act (PDUFA) target action date in the upcoming Day 74 Letter. This expected target date is an important milestone, as it will give both the company and patients a clearer timeline for the regulatory review process of their new treatment. Establishing a definitive review schedule helps in planning subsequent development and potential approval activities.
The announcement comes as part of PTC’s efforts to advance innovative therapies quickly. While the focus is on Huntington’s disease and the promising PTC518 program, the Day 74 Letter represents a key step in meeting the FDA’s rigorous standards. PTC remains committed to collaborating closely with the FDA to ensure that the treatment continues to progress efficiently toward approval.
Read Announcement- Drug:
- Sepiapterin
- Announced Date:
- July 30, 2024
- Indication:
- To treat the broad range of PKU patients.
Announcement
PTC Therapeutics, announced today the submission of the sepiapterin NDA to the U.S. FDA.
AI Summary
PTC Therapeutics has taken a significant step in advancing treatment options for phenylketonuria (PKU) by submitting a New Drug Application (NDA) for sepiapterin to the FDA. This submission targets both pediatric and adult patients with PKU, addressing the full spectrum of the disease. The decision is backed by strong data from the phase 3 APHENITY trial, which demonstrated a notable reduction in blood phenylalanine (Phe) levels—63% overall and 69% in patients with classical PKU. Additionally, the majority of trial participants achieved Phe control as recommended by current guidelines, with some even reaching normalization of levels. The clinical evidence suggests that sepiapterin may allow patients to enjoy less restrictive diets while effectively managing their condition, potentially improving their quality of life. This milestone marks an important advancement in meeting the unmet medical needs of those living with PKU in the U.S.
Read Announcement- Drug:
- Sepiapterin
- Announced Date:
- July 30, 2024
- Indication:
- To treat the broad range of PKU patients.
Announcement
PTC Therapeutics, announced that The European marketing authorization application is currently under review and the marketing authorization applications for Japan and Brazil are expected to be submitted later in 2024.
AI Summary
PTC Therapeutics has announced that its European marketing authorization application for sepiapterin, a promising treatment for phenylketonuria (PKU), is currently under review. This development marks an important step toward making the therapy accessible to patients across different regions.
The company is also planning to expand its global regulatory strategy by preparing to submit marketing authorization applications for Japan and Brazil later in 2024. These efforts highlight PTC Therapeutics’ commitment to addressing the significant unmet needs of both pediatric and adult PKU patients worldwide. By seeking approvals in multiple territories, PTC aims to offer a treatment that may allow patients to manage their condition more effectively and potentially enjoy a more flexible diet, thereby improving their overall quality of life.
Read Announcement- Drug:
- Sepiapterin
- Announced Date:
- May 28, 2024
- Indication:
- To treat the broad range of PKU patients.
Announcement
PTC Therapeutics, Inc. announced today that the sepiapterin MAA for PKU has been validated and accepted for review by the EMA.
AI Summary
PTC Therapeutics announced that its sepiapterin Marketing Authorization Application (MAA) for phenylketonuria (PKU) has been validated and accepted for review by the European Medicines Agency (EMA). This important milestone is a key step toward making sepiapterin available to both children and adults affected by PKU in Europe.
The validation marks the start of a review process that could lead to the approval of sepiapterin, an oral formulation designed to help patients manage their condition better. The application contains data from the phase 3 APHENITY trial, which showed significant reductions in blood phenylalanine levels. PTC plans to make several global submissions in 2024, with this European effort being the first step on a worldwide path to improve treatment options for PKU patients.
Read Announcement
Translarna™ - FDA Regulatory Timeline and Events
Translarna™ is a drug developed by PTC Therapeutics for the following indication: For the treatment of nmDMD.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Translarna™
- Announced Date:
- June 28, 2024
- Indication:
- For the treatment of nmDMD.
Announcement
PTC Therapeutics, Inc. announced that that the CHMP has issued a negative opinion on the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nmDMD.
AI Summary
PTC Therapeutics, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) has issued a negative opinion on renewing the conditional marketing authorization of Translarna™ (ataluren) for treating nonsense mutation Duchenne muscular dystrophy (nmDMD). This negative view follows an earlier negative opinion by the European Commission, which is now under re-review. Despite the setback, the company noted that Translarna’s marketing authorization is still in effect while the re-examination process takes place, and they expect it to remain available through the end of 2024, even if the negative opinion is confirmed.
PTC is disappointed by the decision, especially since the CHMP was asked to review all evidence, including real-world data from the STRIDE study, which many experts believe shows significant efficacy. The company plans to request a formal re-examination to help keep Translarna on the market for affected boys and young men with nmDMD.
Read Announcement
Upstaza - FDA Regulatory Timeline and Events
Upstaza is a drug developed by PTC Therapeutics for the following indication: Aromatic L-amino acid decarboxylase (AADC) deficiency.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Upstaza
- Announced Date:
- May 14, 2024
- Indication:
- Aromatic L-amino acid decarboxylase (AADC) deficiency
Announcement
PTC Therapeutics, Inc. announced that that the FDA has accepted for filing the Biologics License Application (BLA) for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of AADC deficiency. The application has been granted Priority Review with a target regulatory action date of November 13, 2024.
AI Summary
PTC Therapeutics, Inc. announced that the FDA has accepted for filing its Biologics License Application (BLA) for Upstaza™ (eladocagene exuparvovec). Upstaza is a gene therapy developed to treat Aromatic L-Amino Acid Decarboxylase (AADC) deficiency, a rare neurometabolic disorder that can significantly impact motor and cognitive functions.
The application has been granted Priority Review by the FDA, which means the agency will expedite its evaluation process. The target regulatory action date is set for November 13, 2024. This milestone is an important step in PTC Therapeutics’ efforts to provide a new treatment option for patients affected by AADC deficiency, offering hope for improved outcomes in managing this challenging condition.
Read Announcement- Drug:
- Upstaza
- Announced Date:
- May 14, 2024
- Estimated Event Date Range:
- November 13, 2024 - November 13, 2024
- Target Action Date:
- November 13, 2024
- Indication:
- Aromatic L-amino acid decarboxylase (AADC) deficiency
Announcement
PTC Therapeutics, Inc. announced that The application has been granted Priority Review with a target regulatory action date of November 13, 2024.
AI Summary
PTC Therapeutics, Inc. recently announced a significant milestone in its regulatory journey. The company’s application has been granted Priority Review, meaning the FDA will review it faster than usual. This decision is aimed at accelerating the evaluation process because the potential therapy could address important unmet medical needs.
With a target regulatory action date of November 13, 2024, this Priority Review status sets a clear timeline for when a decision might be expected. This fast-track review process shows the FDA's commitment to providing quicker access to potentially life-changing treatments. The news highlights a key moment for PTC Therapeutics as it works to bring its innovative therapy closer to market approval.
Read Announcement
Utreloxastat - FDA Regulatory Timeline and Events
Utreloxastat is a drug developed by PTC Therapeutics for the following indication: In ALS Patients.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Utreloxastat
- Announced Date:
- November 26, 2024
- Indication:
- In ALS Patients
Announcement
PTC Therapeutics, Inc. announced that the global Phase 2 placebo-controlled CardinALS study did not meet its primary endpoint of slowing disease progression on the composite ALSFRS-R and mortality analysis.
AI Summary
PTC Therapeutics, Inc. announced that its global Phase 2, placebo-controlled CardinALS study did not meet its primary endpoint. The trial aimed to slow disease progression in patients with amyotrophic lateral sclerosis (ALS) by measuring changes in the composite ALSFRS-R score along with mortality analysis. Although there was a modest numerical benefit and a trend toward lowering plasma neurofilament light chain—a biomarker of neuronal damage—the results did not reach statistical significance (p=0.52).
The study also failed to achieve its secondary efficacy endpoints. Despite utreloxastat proving to be safe and well tolerated, the lack of efficacy and insufficient biomarker signals have led the company to not plan further development for this treatment. PTC expressed its disappointment and gratitude to all the patients, families, and physicians involved in the study.
Read Announcement
Vatiquinone (MOVE-FA) - FDA Regulatory Timeline and Events
Vatiquinone (MOVE-FA) is a drug developed by PTC Therapeutics for the following indication: Friedreich ataxia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Vatiquinone (MOVE-FA)
- Announced Date:
- February 19, 2025
- Indication:
- Friedreich ataxia
Announcement
PTC Therapeutics, Inc announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich's ataxia (FA).
AI Summary
PTC Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) for vatiquinone, a potential treatment for children and adults with Friedreich’s ataxia (FA). The FDA granted the application Priority Review status and set a Prescription Drug User Fee Act (PDUFA) target action date of August 19, 2025. This decision is an important step toward offering the first approved therapy for pediatric FA patients while also providing a promising treatment alternative for adults. The NDA is supported by data from the placebo-controlled MOVE-FA study and additional long-term studies involving both pediatric and adult FA patients, which showed significant, durable benefits in slowing disease progression and improving key aspects of patient mobility and stability.
Read Announcement- Drug:
- Vatiquinone (MOVE-FA)
- Announced Date:
- February 19, 2025
- Estimated Event Date Range:
- August 19, 2025 - August 19, 2025
- Target Action Date:
- August 19, 2025
- Indication:
- Friedreich ataxia
Announcement
PTC Therapeutics, Inc. announced that PDUFA target action date of Aug. 19, 2025
AI Summary
PTC Therapeutics, Inc. announced that the FDA has accepted its New Drug Application for vatiquinone, a potential treatment for Friedreich’s ataxia in both children and adults. The application has been granted Priority Review, highlighting the significant unmet need for effective treatments in this patient population. A key milestone in this development is the assignment of a Prescription Drug User Fee Act (PDUFA) target action date of August 19, 2025. If approved, vatiquinone would become the first therapy tailored for pediatric patients with Friedreich’s ataxia, while also offering a safe and well-tolerated option for adult patients. The FDA filing was supported by clinical data from the MOVE-FA study and additional long-term trials, which showed promising evidence of slowing disease progression and a favorable safety profile. PTC looks forward to collaborating closely with the FDA during the upcoming review process.
Read Announcement- Drug:
- Vatiquinone (MOVE-FA)
- Announced Date:
- December 19, 2024
- Indication:
- Friedreich ataxia
Announcement
PTC Therapeutics, Inc announced that the submission of the vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich ataxia (FA) to the U.S. Food and Drug Administration (FDA).
AI Summary
PTC Therapeutics, Inc. announced the submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for vatiquinone, a potential treatment for both children and adults living with Friedreich ataxia (FA). If approved, vatiquinone would be the first and only therapy specifically for children suffering from FA. The NDA is supported by data from the placebo-controlled MOVE-FA study and two long-term studies, which showed significant evidence of slowing disease progression and demonstrated that the treatment is safe and well tolerated in all age groups, especially children. Dr. Matthew B. Klein, CEO of PTC Therapeutics, expressed excitement about this milestone, highlighting the potential of vatiquinone to address a significant unmet need among FA patients. This submission marks another step forward in PTC’s efforts to develop transformative therapies for rare disorders.
Read Announcement- Drug:
- Vatiquinone (MOVE-FA)
- Announced Date:
- October 8, 2024
- Indication:
- Friedreich ataxia
Announcement
PTC Therapeutics, Inc. provides several positive updates on the vatiquinone Friedreich ataxia (FA) program.
AI Summary
PTC Therapeutics recently announced positive updates on its vatiquinone program for treating Friedreich’s ataxia (FA). In long-term extension studies, patients receiving vatiquinone demonstrated a significant reduction in disease progression. One study showed that over 144 weeks of treatment, there was a 3.7-point improvement on the modified Friedreich Ataxia Rating Scale (mFARS), representing a 50% slowing of disease progression compared to a natural history cohort. The data confirms that the initial benefits observed in earlier 72-week studies are maintained over a longer treatment period. Additionally, vatiquinone has shown a strong safety profile, with no serious treatment-related adverse events. Bolstered by these positive results, PTC plans to submit a New Drug Application for vatiquinone by December 2024, aiming to provide a meaningful therapy option for FA patients, especially children and adolescents who currently lack approved treatments.
Read Announcement
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