RegulationOrphan Drug (US), Orphan Drug (KR), Orphan Drug (AU), Conditional marketing approval (EU), Priority Review (CN), Fast Track (US), Orphan Drug (EU)

Structure

Molecular FormulaC15H9FN2O3

InChIKeyOOUGLTULBSNHNF-UHFFFAOYSA-N

CAS Registry775304-57-9

Clinical Trials associated with Ataluren

NCT04336826 / CompletedPhase 2

An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy

This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to <2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).

Start Date29 Dec 2021

Sponsor / Collaborator

PTC Therapeutics, Inc.

JPRN-jRCT2041200087 / RecruitingPhase 3IIT

An Open-Label, Long-Term Safety, Efficacy, and Tolerability Study for Ataluren (PTC124) in Patients with Nonsense Mutation Duchenne Muscular Dystrophy - PTC124-GD-049-DMD

Start Date07 Jan 2021

Sponsor / Collaborator-

NCT04014530 / RecruitingPhase 1/2IIT

Study of Pembrolizumab Combined With Ataluren In Patients With Metastatic pMMR and dMMR Colorectal Adenocarcinomas or Metastatic dMMR Endometrial Carcinoma: the ATAPEMBRO Study

Single Center, open label, Phase I-II trial designed to test the safety and efficacy of the combination of Ataluren and Pembrolizumab for the treatment of metastatic mismatch repair deficient and proficient colorectal adenocarcinoma and metastatic mismatch repair deficient endometrial carcinoma.

Start Date01 Aug 2019

Sponsor / Collaborator

Academic Medical Center University of Amsterdam

[+2]

100 Clinical Results associated with Ataluren

100 Translational Medicine associated with Ataluren

100 Patents (Medical) associated with Ataluren

616

Literatures (Medical) associated with Ataluren

01 Dec 2024·Molecular Genetics and Metabolism Reports

Ataluren-mediated nonsense variant readthrough in D-bifunctional protein deficiency: A case report

Article

Author: Lee, Ni-Chung ; Hwu, Wuh-Liang ; Chien, Yin-Hsiu ; Lee, Wang-Tso ; Hsu, Rai-Hseng ; Chen, Hui-An

D-bifunctional protein (DBP) deficiency, a fatal peroxisomal enzyme disorder, typically manifests with life-threatening symptoms in the first two years of childhood. We present the case of an infant with elevated lysophosphatidylcholine C26:0 (C26:0-LPC) levels identified during X-linked adrenoleukodystrophy (ALD) screening, leading to a diagnosis of DBP deficiency due to a homozygous HSD17B4 c.1041T>A, p.(Tyr347Ter) variant. Starting at two months of age, the infant experienced seizures, hypotonia, and developmental delays, prompting the initiation of experimental treatment with the readthrough agent PTC124 (ataluren) at six months. The treatment led to a decrease in C26:0-LPC levels from 0.65 μM to 0.53 μM; concomitant fish oil supplementation transiently increased C26:0-LPC to 0.74 μM before returning to 0.53 μM after cessation of supplementation. The patient demonstrated improved swallowing and progressive motor and speech development during a two-year treatment period, with no further seizures. This case report highlights the potential of nonsense readthrough therapy for peroxisomal disorders, a group of metabolic diseases that currently lack targeted treatments.

01 Oct 2024·EXPERIMENTAL NEUROLOGY

Investigating therapeutic nonsense suppression in a neurofibromatosis mouse model

Article

Author: Peker, Ahmet ; Wu, Chan ; Shazeeb, Mohammed Salman ; Han, George ; Gounis, Matthew J ; Jacobson, Allan ; Mangkalaphiban, Kotchaphorn ; Rentiya, Zubir S

Neurofibromatosis type 1 (NF1) is a human genetic disorder caused by variants in the NF1 gene. Plexiform neurofibromas, one of many NF1 manifestations, are benign peripheral nerve sheath tumors occurring in up to 50% of NF1 patients. A substantial fraction of NF1 pathogenetic variants are nonsense mutations, which result in the synthesis of truncated non-functional NF1 protein (neurofibromin). To date, no therapeutics have restored neurofibromin expression or addressed the consequences of this protein's absence in NF1 nonsense mutation patients, but nonsense suppression is a potential approach to the problem. Ataluren is a small molecule drug that has been shown to stimulate functional nonsense codon readthrough in several models of nonsense mutation diseases, as well as in Duchenne muscular dystrophy patients. To test ataluren's potential applicability in nonsense mutation NF1 patients, we evaluated its therapeutic effects using three treatment regimens in a previously established NF1 patient-derived (c.2041C > T; p.Arg681X) nonsense mutation mouse model. Collectively, our experiments indicate that: i) ataluren appeared to slow the growth of neurofibromas and alleviate some paralysis phenotypes, ii) female Nf1-nonsense mutation mice manifested more severe paralysis and neurofibroma phenotypes than male mice, iii) ataluren doses with apparent effectiveness were lower in female mice than in male mice, and iv) age factors also influenced ataluren's effectiveness.

01 Oct 2024·PEST MANAGEMENT SCIENCE

Design, synthesis, antimicrobial activity, and mechanism of novel 3‐(2,4‐dichlorophenyl)‐[1,2,4]triazolo[3,4‐b][1,3,4]thiadiazole derivatives

Article

Author: Zhang, Renfeng ; Xue, Wei ; Chen, Mei ; Zheng, Yuguo

Abstract:

BACKGROUND:

Plant pathogens cause substantial crop losses annually, posing a grave threat to global food security. Fungicides have usually been used for their control, but the rapid development of pesticide resistance renders many ineffective, therefore the search for novel and efficient green pesticides to prevent and control plant diseases has become the top priority in crop planting.

RESULTS:

The results of bioassay studies indicated that most of the target compounds showed certain antimicrobial activity in vitro. In particular, compound X7 showed high inhibitory activity against Xanthomonas oryzae pv. oryzae (Xoo), with an EC50 value of 27.47 μg mL−1, surpassing conventional control agents such as thiazole zinc (41.55 μg mL−1) and thiodiazole copper (53.39 μg mL−1). Further studies on molecular docking showed that X7 had a strong binding affinity with 2FBW. The morphological change observed by scanning electron microscopy indicated that the surface of Xoo appears wrinkled and cracked under X7 treatment and a total of 2662 proteins were identified by label‐free proteomic analysis. Three experiments have elucidated the mechanism whereby X7 induced considerable changes in the physiological and biochemical properties of Xoo, which in turn affected the reproduction and growth of bacteria.

CONCLUSION:

News (Medical) associated with Ataluren

30 Oct 2024

·www.prnewswire.com

PTC Therapeutics Announces FDA Acceptance of Translarna™ NDA Resubmission

WARREN, N.J., Oct. 30, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the New Drug Application (NDA) for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). "The NDA acceptance for review is a significant milestone that brings us one step closer to providing this important treatment to boys and young men living with nonsense mutation Duchenne muscular dystrophy in the United States," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. "The totality of evidence clearly supports the favorable safety profile and short- and long-term benefits of Translarna for individuals with nmDMD. We look forward to working with FDA throughout the review process." "We thank FDA for accepting the Translarna NDA for review," stated Pat Furlong, Founder and CEO, Parent Project Muscular Dystrophy. "Translarna uniquely addresses individuals with Duchenne muscular dystrophy secondary to nonsense mutation and could provide an important treatment option for our community. Families in the United States have been waiting a long time for a treatment that targets the underlying cause of nonsense mutation Duchenne." The NDA resubmission is based on the findings of significant benefit demonstrated in the ITT population (N=359) of the global placebo-controlled trial Study 041. Following 72-weeks of Translarna treatment, there was significant benefit demonstrated on the key study endpoints of six-minute walk distance (6MWD) (p=0.0248), NorthStar Ambulatory Assessment (p=0.0283), 10-meter walk/run (p=0.0422), 4-stair climb (p=0.0293), and time to 10% worsening of 6MWD (p=0.0078). In addition, the NDA includes the findings of significant long-term Translarna treatment benefit as captured in the STRIDE registry. Translarna treatment resulted in a 3.5-year delay in loss of ambulation (p<0.0001) and a 1.8-year delay in reaching a predicted forced vital capacity of less than 60% (p=0.0028) a critical threshold of lung function. "We are excited that the FDA has accepted the Translarna NDA for review," said Debra Miller, Founder and CEO of CureDuchenne. "We believe that the totality of the data demonstrates the meaningful benefits and strong safety profile of Translarna for people with Duchenne muscular dystrophy caused by a nonsense mutation, which is approximately 13% of our community. Many of our boys and young men have participated in Translarna clinical trials over the years, and about 150 of them remain on therapy through extension studies and continue to experience the benefits of Translarna, including maintaining independence." As this was an NDA resubmission following a complete response letter to the NDA which was filed over protest in 2016, FDA is not obligated to follow PDUFA review timelines. Thus, an action date has not been provided. About Translarna™ (ataluren) Translarna (ataluren), discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne. Translarna, the tradename of ataluren, is licensed in multiple countries. Study 041 was a global placebo-controlled trial that enrolled a broad population of nmDMD patients. The study included a primary analysis population of a subgroup of enrollees, which did not reach statistical significance on the primary endpoint of 6MWD. However, significant benefit was recorded across 6MWD and other key study endpoints in the overall enrolled (ITT) population. About the STRIDE Registry The STRIDE Registry is an ongoing, multicenter, observational study of the safety and effectiveness of Translarna in routine care. It is the first patient data repository to provide real-world experience regarding the long-term use of Translarna in routine clinical practice. STRIDE is a collaborative partnership between TREAT-NMD and PTC Therapeutics, led by a Steering Committee comprised of leading experts in Duchenne, patient advocates from around the world and PTC representatives. About Duchenne Muscular Dystrophy (Duchenne) Primarily affecting males, Duchenne is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-20's due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk (loss of ambulation) as early as 10 years old, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and 20s. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at and follow us on Facebook, Instagram, LinkedIn and Twitter at @PTCBio. For More Information: Investors: Investor Relations +1 (908) 912-9848 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statement: This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of regulatory submissions and responses, commercialization and other matters with respect to its products and product candidates; PTC's plans for interactions with the U.S. Food and Drug Administration (FDA); the clinical utility and potential advantages of Translarna (ataluren); PTC's strategy, future operations, future financial position, future revenues, projected costs; the extent, timing and financial aspects of our strategic pipeline prioritization and reductions in workforce; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia, the European Economic Area (EEA) and other regions, or PTC's ability to identify other potential mechanisms by which it may provide Translarna to nmDMD patients in the EEA; PTC's ability to use the clinical data from its international drug registry study and real-world evidence concerning Translarna's benefits to support a continued marketing authorization for Translarna for the treatment of nmDMD in the EEA; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from PTC's trials in Translarna; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; and the factors discussed in the "Risk Factors" section of PTC's most recent Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Translarna. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

NDAClinical Result

22 Oct 2024

·www.outsourcing-pharma.com

PTC Therapeutics’ Translarna misses European regulatory hurdle again

The EU first gave Translarna a conditional marketing authorization in 2014 for the treatment of DMD, a progressive condition that causes the muscles to weaken, eventually becoming life-threatening. Eligible patients for the treatment are at least 2 years old, are able to walk, and their disease is caused by a so-called nonsense mutation in the protein dystrophin, which strengthens and protects muscle fibers The initial approval decision was weighted by a lack of treatments for DMD and was contingent on further trials to confirm its benefits. However, two studies of Translarna after the authorization called 020 and 041 missed their efficacy goals. While a specific subgroup of patients in study 020 did show evidence of responding to the drug, patients in 041 saw no significant differences in the deterioration of their walking ability relative to those in the placebo group. The CHMP produced a negative opinion for Translarna in September 2023, which it confirmed in January 2024. The CHMP then went back into discussion in May this year when the European Commission requested that the committee consider the evidence in addition to more real-world data, but upheld the rejection. In July this year, PTC Therapeutics requested a re-evaluation of the treatment from the studies in addition to data from a different study comparing patients from different registries, which appeared to show a delay in disability in some treated patients. However, the CHMP once again declined, citing differences between the registries and biases. It also took into account third-party perspectives from patients and healthcare professionals. googletag.cmd.push(function () { googletag.display('text-ad1'); }); In a public statement , Matthew Klein, CEO of PTC Therapeutics, said the "CHMP again based its decision on the results of the primary analysis subpopulation of Study 041 instead of the totality of evidence for Translarna.” He later said that the decision “is clearly against the expressed wishes of physicians, patients and families throughout Europe.” The opinion will now be reviewed by the European Commission, which will make a legally binding decision within around two months. For now, Translarna is still commercially available and PTC is working to make sure the EC has the evidence it needs. Klein told Outsourcing Pharma that in discussions with FDA, the US regulator has been open to considering review of an NDA based on the totality of evidence, unlike the EMA’s CHMP. The DMD landscape There is no cure for DMD, with current treatments including surgery to correct postural deformities and steroids to improve muscle strength. Translarna is designed to restore the production of dystrophin in muscles by making cells bypass the nonsense mutation when making the protein. The market for DMD treatments was projected to grow from $3.42 billion in 2024 to $8.19 billion by 2029, with a growth of 19.13% per year. The growth is driven by the increasing DMD burden and rising investments in the space. Translarna itself bagged $355.8 million in net revenues in 2023 compared to $288.6 million in 2022. Other treatments on the market for various types of DMD include Sarepta’s AMONDYS 45™ (casimersen) and gene therapy Elevidys. Meanwhile, Pfizer’s gene therapy fordadistrogene movaparvovec hit a setback in a phase 3 trial when it failed to meet its primary efficacy endpoint. “DMD is a highly morbid, complex and rare disorder that will continue to be treated with a combination of therapies, including those that target inflammation (such as deflazacort or other corticosteroids) and genetic targeted therapies, such as Translarna in the case of DMD secondary to nonsense mutations,” Klein said. “Given disease heterogeneity it will be important for there to be multiple therapeutic options.”

Phase 3Drug ApprovalGene Therapy

18 Oct 2024

·www.prnewswire.com

CHMP Maintains Negative Opinion on Translarna™ Reexamination

- Opinion to be reviewed by European Commission - WARREN, N.J., Oct. 18, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has maintained its negative opinion on the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne Muscular Dystrophy (nmDMD) following re-examination. The opinion will now be reviewed by the European Commission (EC) which is expected to decide on opinion adoption in approximately 67 days. "CHMP again based its decision on the results of the primary analysis subpopulation of Study 041 instead of the totality of evidence for Translarna. That evidence includes data across three placebo-controlled trials and our STRIDE registry that demonstrates consistent short and long-term efficacy as well as safety supportive of continued authorization. In addition, the CHMP opinion is clearly against the expressed wishes of physicians, patients and families throughout Europe," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics, Inc. "As Translarna will remain authorized in Europe pending review by the European Commission, we will continue to ensure Translarna is available to boys and young men living with nonsense mutation Duchenne muscular dystrophy. In addition, PTC will ensure the EC has all possible evidence to support continued authorization." About Translarna™ (ataluren) Translarna (ataluren), discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne. Translarna, the tradename of ataluren, is licensed in multiple countries for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged 2 years and older. Ataluren is an investigational new drug in the United States. About Duchenne Muscular Dystrophy (Duchenne) Primarily affecting males, Duchenne is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-20's due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk (loss of ambulation) as early as 10 years old, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and 20s. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at and follow us on X, Facebook, Instagram and LinkedIn. For More Information: Investors: Investor Relations +1 (908) 912-9848 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statement This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, commercialization and other matters with respect to its products and product candidates; PTC's plans for interactions with the European Medicines Agency (EMA); the European Commission's potential adoption of the CHMP's negative opinion for Translarna (ataluren); the clinical utility and potential advantages of Translarna (ataluren); PTC's strategy, future operations, future financial position, future revenues, projected costs; the extent, timing and financial aspects of our strategic pipeline prioritization and reductions in workforce; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia, the European Economic Area (EEA) and other regions, or PTC's ability to identify other potential mechanisms by which it may provide Translarna to nmDMD patients in the EEA; PTC's ability to use the clinical data from its international drug registry study and real-world evidence concerning Translarna's benefits to support a continued marketing authorization for Translarna for the treatment of nmDMD in the EEA; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from PTC's trials in Translarna; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; and the factors discussed in the "Risk Factors" section of PTC's most recent Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Translarna. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug Approval

100 Deals associated with Ataluren

External Link

KEGG	Wiki	ATC	Drug Bank
D09323	Ataluren	M09AX03	DB05016

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Muscular Dystrophy, Duchenne	EU	PTC Therapeutics International Ltd.	31 Jul 2014
Muscular Dystrophy, Duchenne	IS	PTC Therapeutics International Ltd.	31 Jul 2014
Muscular Dystrophy, Duchenne	LI	PTC Therapeutics International Ltd.	31 Jul 2014
Muscular Dystrophy, Duchenne	NO	PTC Therapeutics International Ltd.	31 Jul 2014

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Cystic Fibrosis	NDA/BLA	EU	PTC Therapeutics, Inc.	-
Becker Muscular Dystrophy	Phase 3	AU	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	BE	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	CA	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	FR	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	DE	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	IL	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	IT	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	ES	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	SE	PTC Therapeutics, Inc.	20 May 2012

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04336826 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	6	Ataluren	hphfumhdgs(vemojcowfg) = gfsmuhczdb qjtnjzhwer (qklpofrczu, fhwgkppznk - cmxcmudshd) View more	-	01 Apr 2024
NCT03179631 \| NCT01826487 \| NCT00592553 (Study 041 \| Study 007 \| ACT DMD, MDA2024)	Phase 2/3	Muscular Dystrophy, Duchenne	-	Ataluren	yajocczpbt(lpajlsfeps) = evimtumkns yucaxdliiz (ktptyiuvew ) View more	Positive	03 Mar 2024
NCT03179631 (Study 041, AAN2023)	Phase 3	Muscular Dystrophy, Duchenne nonsense mutation (nm) DMD	-	Ataluren	ukdbmhbxwq(uamrmblzdd) = mvvugahagn kxzlxcweiq (isgrotwfip ) View more	Positive	25 Apr 2023
NCT03179631 (Study 041, MDA2023)	Phase 3	Muscular Dystrophy, Duchenne	-	Ataluren	mzsxxabxxu(rqvkolwznn) = olxcqkzlnk yfmhzlsdzc (xqukxljwlk ) View more	Positive	19 Mar 2023
NCT03179631 (Study 041, MDA2023)	Phase 3	Muscular Dystrophy, Duchenne	701	Ataluren	(jiqpgeymzg) = aidmualsio ltznpkyvlj (wzhpkrosjd ) View more	Positive	19 Mar 2023
Pubmed	Not Applicable	Cystic Fibrosis	517	Ataluren and similar compounds	fpkzhuvzkl(siveuamhiv) = Ataluren was associated with a higher rate of episodes of renal impairment (risk ratio 12.81, 95% confidence interval 2.46 to 66.65; P = 0.002; I2 = 0%; 2 trials, 517 participants) mytkcoarke (peipjlvnhs ) View more	-	03 Mar 2023
Pubmed	Not Applicable	Cystic Fibrosis	517	Placebo		-	03 Mar 2023
NCT02647359 (STAR, CTgov)	Phase 2	Aniridia	39	Ataluren (Ataluren)	dpvqhmdzzz(mvryyzkmkt) = rldszjurbw pysqkhfqjn (gqkgrzfwrv, rcdudsnvbr - soiaxenrqo) View more	-	27 May 2022
NCT02647359 (STAR, CTgov)	Phase 2	Aniridia	39	Placebo+Ataluren (Placebo)	dpvqhmdzzz(mvryyzkmkt) = xjcjwijsno pysqkhfqjn (gqkgrzfwrv, ezilrhmsce - qrtrurjqoe) View more	-	27 May 2022
NCT03648827 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	20	Ataluren	bpmvugpnph(guxdhbodhy) = ucolmxnlaa gtnefdfele (estpsqejxi, vcayajrudc - fzfioqjygs) View more	-	05 Apr 2022
NCT03796637 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	6	Ataluren	mbukubjdjx(rbahamowtl) = rflafxujhd svuzymylzq (xpcvuzuvpu, fxieedmccx - bjzxtmgnwq) View more	-	05 Apr 2022
NCT01557400 (Study 019, MDA2021)	Phase 3	Muscular Dystrophy, Duchenne nonsense mutation	94	Ataluren 10, 10, 20 mg/kg	scntferlqn(vkbrgfxdfk) = a trend in delay in decline to predicted FVC <50% by ~1 year zegehzmzlo (ohgdvmiijb )	Positive	01 Mar 2021
NCT01557400 (Study 019, MDA2021)	Phase 3	Muscular Dystrophy, Duchenne nonsense mutation	94	Ataluren		Positive	01 Mar 2021

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