X
Pharma Advancement
  • Home
  • Drug Development
    • All
    • Clinical Trials
    • FDA Approvals
    • Research & Development
    development of new antibiotics

    EMA guidance supports development of new antibiotics

    Thermo Fisher Scientific Announces Collaboration with Northeastern University to Advance Biopharmaceutical Characterization and Monitoring Workflows

    Commercial Success In New Biopharmaceutical Innovation Era

    Proteros and AstraZeneca Boost Collaboration Agreement

    Conver to wp

    AbbVie and Cugene Join Forces Concerning Autoimmune Disorders and Cancer

    Drug Imagent For Therapeutic Platform

    Japan Pharma Lobby Says-Price Scheme Is Causing Drug Lag

    Charles River and Valence Discovery Announce Strategic Partnership to Provide Clients with AI-Enabled Drug Design Capabilities

    Charles River and Valo Launch Logica, an Integrated AI-Powered Drug Discovery Solution to Rapidly Deliver Optimized Preclinical Assets

    AstraZeneca announces phase IV ASCENT trial of Tudorza Pressair in patients with COPD meets primary efficacy endpoint

    AstraZeneca announces plans for new strategic R&D centre and Alexion headquarters in Cambridge, Massachusetts

    Lynparza approved in the EU for BRCA-mutated metastatic pancreatic cancer

    Major Success Factors For Clinical Development Organizations

    ArisGlobal Expands LifeSphere Clinical Portfolio with Landmark Customer Wins in APAC & Middle East

    ArisGlobal Expands LifeSphere Clinical Portfolio with Landmark Customer Wins in APAC & Middle East

  • Manufacturing
  • Supply Chain
  • Facilities
  • Health & Nutrition
  • Events
  • Contact Us
No Result
View All Result
  • Home
  • Drug Development
    • All
    • Clinical Trials
    • FDA Approvals
    • Research & Development
    development of new antibiotics

    EMA guidance supports development of new antibiotics

    Thermo Fisher Scientific Announces Collaboration with Northeastern University to Advance Biopharmaceutical Characterization and Monitoring Workflows

    Commercial Success In New Biopharmaceutical Innovation Era

    Proteros and AstraZeneca Boost Collaboration Agreement

    Conver to wp

    AbbVie and Cugene Join Forces Concerning Autoimmune Disorders and Cancer

    Drug Imagent For Therapeutic Platform

    Japan Pharma Lobby Says-Price Scheme Is Causing Drug Lag

    Charles River and Valence Discovery Announce Strategic Partnership to Provide Clients with AI-Enabled Drug Design Capabilities

    Charles River and Valo Launch Logica, an Integrated AI-Powered Drug Discovery Solution to Rapidly Deliver Optimized Preclinical Assets

    AstraZeneca announces phase IV ASCENT trial of Tudorza Pressair in patients with COPD meets primary efficacy endpoint

    AstraZeneca announces plans for new strategic R&D centre and Alexion headquarters in Cambridge, Massachusetts

    Lynparza approved in the EU for BRCA-mutated metastatic pancreatic cancer

    Major Success Factors For Clinical Development Organizations

    ArisGlobal Expands LifeSphere Clinical Portfolio with Landmark Customer Wins in APAC & Middle East

    ArisGlobal Expands LifeSphere Clinical Portfolio with Landmark Customer Wins in APAC & Middle East

  • Manufacturing
  • Supply Chain
  • Facilities
  • Health & Nutrition
  • Events
  • Contact Us
No Result
View All Result
Pharma Advancement
No Result
View All Result
Home Americas

Stealth BioTherapeutics Granted Fast Track Designation For The Treatment Of Barth Syndrome

Yuvraj_pawp by Yuvraj_pawp
28th November 2017
in Americas, News

Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its lead candidate, elamipretide, for the treatment of Barth syndrome.

“Fast Track designation is an important milestone which will facilitate Stealth’s efforts to develop an effective treatment for the Barth syndrome patient community, for whom there are currently no FDA-approved therapies,” said Stealth Chief Executive Officer Reenie McCarthy. “We are committed to developing treatments for patients suffering from rare mitochondrial diseases such as Barth syndrome, and look forward to working closely with the FDA in addressing this critical unmet need.”

In July 2017, Stealth initiated TAZPOWER, a Phase 2/3, randomized, double-blind, placebo-controlled crossover study that will evaluate the effects of daily treatment with elamipretide in patients with genetically confirmed Barth syndrome. Top-line results from the study are expected in 2018.

Barth syndrome is a rare genetic mitochondrial disease, caused by mutations in the TAZ gene. The disease is characterized by skeletal muscle weakness, cardiac abnormalities often leading to heart failure, recurrent infections, delayed growth and reduced life expectancy. There are currently no FDA-approved therapies for the disease.

Elamipretide was previously granted Fast Track designation for the treatment of primary mitochondrial myopathy (PMM), another rare mitochondrial disease, in December 2015. The FDA’s Fast Track program facilitates the development and review of drugs to treat serious conditions with unmet medical needs. The designation provides the opportunity for more frequent meetings with the FDA over the course of development, and allows a drug company to submit individual sections of its New Drug Application (NDA) for review as they are ready, rather than waiting until all sections of the NDA are complete. Further, the designation also increases the likelihood of eligibility for priority review and accelerated approval if relevant criteria are met.

For additional information on the TAZPOWER study or elamipretide, please refer to Stealth’s website.

About Barth Syndrome
Barth syndrome is a rare genetic condition characterized by muscle weakness, cardiac abnormalities often leading to heart failure, recurrent infections, delayed growth, and reduced life expectancy. Barth syndrome occurs almost exclusively in males and is estimated to affect one in 200,000 to 400,000 individuals worldwide at birth. There are currently no FDA-approved therapies for the disease.

About Stealth
We are a privately held clinical-stage biotechnology company focused on the development of therapeutics for diseases involving mitochondrial dysfunction. We believe there is a strong rationale for our lead product candidate, elamipretide, in indications in these diseases based on encouraging preclinical and early clinical data. We are investigating elamipretide in three primary mitochondrial diseases – PMM, Barth syndrome and Leber’s hereditary optic neuropathy – as well as in heart failure and dry age-related macular degeneration. We received Orphan Drug Designation of elamipretide for the treatment of PMM from the FDA in September 2017. We are developing our second product candidate, SBT-20, for neurodegenerative diseases. To learn more information about us and our pipeline, visit www.stealthbt.com

Contacts
Media Relations
dna Communications
Kate Contreras, 617-520-7088
Media@StealthBT.com

Investor Relations
Stern IR
Beth DelGiacco, 212-362-1200
IR@StealthBT.com

Previous Post

Bristol-Myers Squibb Awards “Golden Tickets” for LabCentral to Neutrolis, Remora Therapeutics

Next Post

Innovest Global Inc. Signs Letter Of Intent to Acquire Commercial Energy Business

Related Posts

Dupixent From Sanofi Delivers Another Fantastic Performance
Manufacturing

Sanofi Sets Up Fund To Enable Firms In Low-Income Countries

5th July 2022
GSK commences phase III study of Benlysta and rituximab combination in systemic lupus erythematosus
Manufacturing

Singapore Authorises New Ovarian Cancer Treatment By GSK

5th July 2022
Abbott Is Named Global Industry Leader in Sustainability for the Sixth Consecutive Year on the Dow Jones Sustainability Index (DJSI)
Manufacturing

Abbott India Affirms That The Medicines Are Not Their Own

5th July 2022
Global Biopharmaceutical Bioseparation Market Eyeing $20bn
Manufacturing

Global Biopharmaceutical Bioseparation Market Eyeing $20bn

4th July 2022
FDA Advises Adding Omicron BA.4, 5 To COVID-19 Boosters
Manufacturing

FDA Advises Adding Omicron BA.4, 5 To COVID-19 Boosters

4th July 2022
COVID-19 Omicron-Specific Vaccines Could Be Used As Boosters
Manufacturing

COVID-19 Omicron-Specific Vaccines Could Be Used As Boosters

4th July 2022
Next Post
Innovest Global Inc. Signs Letter Of Intent to Acquire Commercial Energy Business

Innovest Global Inc. Signs Letter Of Intent to Acquire Commercial Energy Business

Latest News

Dupixent From Sanofi Delivers Another Fantastic Performance
Manufacturing

Sanofi Sets Up Fund To Enable Firms In Low-Income Countries

5th July 2022
GSK commences phase III study of Benlysta and rituximab combination in systemic lupus erythematosus
Manufacturing

Singapore Authorises New Ovarian Cancer Treatment By GSK

5th July 2022
Abbott Is Named Global Industry Leader in Sustainability for the Sixth Consecutive Year on the Dow Jones Sustainability Index (DJSI)
Manufacturing

Abbott India Affirms That The Medicines Are Not Their Own

5th July 2022
Global Biopharmaceutical Bioseparation Market Eyeing $20bn
Manufacturing

Global Biopharmaceutical Bioseparation Market Eyeing $20bn

4th July 2022
FDA Advises Adding Omicron BA.4, 5 To COVID-19 Boosters
Manufacturing

FDA Advises Adding Omicron BA.4, 5 To COVID-19 Boosters

4th July 2022
Pharma Advancement

About Us

Pharma Advancement is a leading Pharma information centric website. On one side Pharmaadvancement.com has established itself as one of the most efficient and comprehensive source of Pharma information online, dedicated to providing decision makers in all the Pharma industry sectors with reliable, accurate and useful insights into happenings in the Pharma sector.

Qucik Links

  • Drug Development
  • Manufacturing
  • News
  • Events & Conferences
  • Newsletter Archive

Resources

  • Advertise with us
  • Contact Us
  • Download Mediapack

System

  • Search
  • Sitemap
  • RSS Feed

© 2017 Copyright © Valuemediaservices 2017 All rights reserved.

No Result
View All Result
  • Home
  • Drug Development
  • Manufacturing
  • Supply Chain
  • Facilities
  • Health & Nutrition
  • Events
  • Contact Us

© 2017 Copyright © Valuemediaservices 2017 All rights reserved.

Login to your account below

Forgotten Password?

Fill the forms bellow to register

All fields are required. Log In

Retrieve your password

Please enter your username or email address to reset your password.

Log In