Breaking News
October 16, 2018 - Poor Outcomes for Hispanic Infants With Congenital Heart Dz
October 16, 2018 - Global study finds youngest in class more likely to be diagnosed with ADHD
October 16, 2018 - Researchers sequence two selfish genes in the fungus Neurospora intermedia
October 16, 2018 - Survey results highlight the need for better communication between patients and HCPs about bacterial vaginosis
October 16, 2018 - Researchers develop fibrin-targeting immunotherapy to protect against neurodegeneration
October 16, 2018 - Researchers create open access database on healthy immunity
October 16, 2018 - Rice University chemist wins big award to study small surfaces
October 16, 2018 - Study finds 43% drop in stroke rate
October 16, 2018 - Researchers identify basic relationships of cell cycle and cellular senescence in the placenta
October 16, 2018 - Obesity Doubles Odds for Colon Cancer in Younger Women
October 16, 2018 - Adults with ADHD not constrained in creativity
October 16, 2018 - Raising visibility for people and students with chronic illness and disability
October 16, 2018 - Allele awarded NIH grant to develop nanoantibody therapies for treatment of sepsis
October 16, 2018 - Only 59% of young adults undergoing surgery are fluid responsive
October 16, 2018 - Research points to potential new treatment for hearing loss
October 16, 2018 - MDI Biological Laboratory receives $1.2 million SEPA grant to promote data literacy
October 16, 2018 - Vast majority of dementia cases may arise from spontaneous genetic errors
October 16, 2018 - New project aims to deliver fast, effective treatment for autoimmune rheumatic diseases
October 16, 2018 - Study identifies molecular switch that controls fate of milk-producing breast cells
October 16, 2018 - Research shows diet has little influence on precursor to gout
October 16, 2018 - “Without Dr. Shumway doing his miracle work, three generations would not be here”: A Stanford heart transplant patient’s story
October 16, 2018 - Non-invasive brain stimulation sheds light on neurobiology underlying implicit bias
October 16, 2018 - Researchers demonstrate integrated technique to control production of cell therapeutics
October 16, 2018 - Breast tomosynthesis detects 34% more tumors than traditional mammography
October 16, 2018 - Rhode Island Hospital, Brown receive $800,000 grant to keep up fight against opioid epidemic
October 16, 2018 - UVA partners with health systems in AVIA network’s Medicaid Transformation Project
October 16, 2018 - Trevena Announces Oliceridine FDA Advisory Committee Meeting Outcome
October 16, 2018 - Study reveals early warning signs of heart problems in patients with newly diagnosed lupus
October 16, 2018 - Connecting the dots of Alzheimer’s disease
October 16, 2018 - New publication offers evidence-based content for global breast imaging medical community
October 16, 2018 - ‘EinsteinVision’ that improves hand-eye coordination of surgeons introduced at Harefield Hospital
October 16, 2018 - WRAIR clinical study evaluates safety and immunogenicity of Marburg vaccine
October 16, 2018 - Ketamine can be considered as alternative to opioids for short-term pain control in ED
October 16, 2018 - Endurance exercise training beneficially alters gut microbiota composition
October 15, 2018 - FDA Approves Yutiq (fluocinolone acetonide intravitreal implant) for Chronic Non-Infectious Posterior Segment Uveitis
October 15, 2018 - Birthing Options for Full-Term Pregnancy
October 15, 2018 - Stressed, toxic, zombie cells seen for first time in Alzheimer’s
October 15, 2018 - Concussion researchers study head motion in high school football hits | News Center
October 15, 2018 - Neuropsychiatric symptoms related to earliest stages of Alzheimer’s brain pathology
October 15, 2018 - Neck collar device may help protect the brain of female high school soccer players
October 15, 2018 - Research reveals how the inner ear processes speech
October 15, 2018 - Many parents still skeptical about safety and effectiveness of flu shot, survey finds
October 15, 2018 - Payer Policies May Discourage Non-Pharma Tx for Low Back Pain
October 15, 2018 - Exercise may delay cognitive decline in people with rare Alzheimer’s disease
October 15, 2018 - Researchers modify CRISPR to reorganize genome | News Center
October 15, 2018 - Innovative brain tumor operation set to tailor to patients’ needs
October 15, 2018 - Findings offer new insight into early changes that occur during AD pathology
October 15, 2018 - Neurons regulating reproductive hormone release have different activity in epileptic mice
October 15, 2018 - More parents are concerned about taking babies swimming in public pools
October 15, 2018 - Health Tip: Know the Risk Factors for Lower Back Pain
October 15, 2018 - Study shows cigarillo flavors enhanced by high-intensity sweeteners
October 15, 2018 - Study traces hospital-acquired bloodstream infections to patients’ own bodies | News Center
October 15, 2018 - Abnormal vision in childhood can affect development of brain areas responsible for attention
October 15, 2018 - Study highlights need for increased support for alcohol-related liver disease patients
October 15, 2018 - Color-changing contact lens could help doctors to monitor eye disease medications
October 15, 2018 - Tobacco heating products cause less staining to teeth than conventional cigarettes
October 15, 2018 - Young adults who are obese can expect to lose up to 10 years in life expectancy
October 15, 2018 - Scientists uncover how proteins meet on the cell membrane
October 15, 2018 - Affordable housing with supportive social services for senior citizens can reduce hospital use
October 15, 2018 - Schiller Easy Pulse Saves Lives
October 15, 2018 - The latest ECG device from Schiller
October 15, 2018 - Following a Tissue Sample
October 15, 2018 - Prisoners need drug and alcohol treatments but AA programs aren’t the answer
October 15, 2018 - Andrea Califano and Jordan Orange Elected to National Academy of Medicine
October 15, 2018 - The impending risk of African Swine Fever Virus
October 15, 2018 - Breastfeeding reduces the number of antibiotic-resistant bacteria in infant gut
October 15, 2018 - Researchers develop comprehensive molecular atlas of postnatal mouse heart development
October 15, 2018 - ObsEva SA Presents Clinical Data from Phase III IMPLANT 2 Trial of Nolasiban in IVF at the American Society of Reproductive Medicine (ASRM) Annual Meeting
October 15, 2018 - Engineering teratoma-derived fibroblasts to enhance osteogenesis
October 15, 2018 - Lab study shows effectiveness of potential therapy for treatment-resistant hypothyroidism
October 15, 2018 - JCU study firms up association between diet and depression
October 15, 2018 - Researchers to study the use of CRISPR on human liver on-a-chip platform
October 15, 2018 - Sub-concussive impacts not associated with decline in neurocognitive function
October 15, 2018 - Researchers find potential treatment to halt premature labor and birth
October 15, 2018 - As U.S. suicides rates rise, Hispanics show relative immunity
October 15, 2018 - FDA Issues a Complete Response Letter to Acacia Pharma for Barhemsys
October 15, 2018 - Photoactive bacteria bait may help in fight against MRSA infections
October 15, 2018 - Increasing vigorous exercise reduces risk factors of type 2 diabetes, cardiovascular disease in children
October 15, 2018 - First-of-its-kind study to test a personalized vaccine in cancer patient
October 15, 2018 - Extension trial assesses benefit of switching from flash monitoring to RT-CGM for hypoglycemia
Stealth BioTherapeutics Granted Fast Track Designation for Elamipretide for the Treatment of Leber’s Hereditary Optic Neuropathy

Stealth BioTherapeutics Granted Fast Track Designation for Elamipretide for the Treatment of Leber’s Hereditary Optic Neuropathy

image_pdfDownload PDFimage_print

BOSTON – December 18, 2017 – 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 Leber’s hereditary optic neuropathy (LHON).

“We are pleased with the FDA’s decision to grant Fast Track designation to elamipretide for the potential treatment of LHON,” Stealth BioTherapeutics Chief Executive Officer Reenie McCarthy said. “This designation, a first for our ophthalmology program, is evidence of the serious need for new therapies for those suffering from this devastating rare mitochondrial disease.”

LHON is an inherited mitochondrial disease that causes loss of central vision due to damage to neurons in the retina, called retinal ganglion cells, as a result of dysfunctional mitochondria. The disease, which affects approximately 35,000 people worldwide, can lead to legal blindness.

In June 2016, Stealth announced the initiation of ReSIGHT, a Phase 2, prospective, double-masked, vehicle-controlled clinical study to evaluate the safety, tolerability and efficacy of topical eye drop delivery of elamipretide in people with LHON. Top-line results from the study are expected by mid-2018.

Elamipretide has also been granted Fast Track designation for the treatment of primary mitochondrial myopathy (PMM) and Barth syndrome, two rare primary mitochondrial diseases.

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. The designation also increases the likelihood of eligibility for priority review and accelerated approval if relevant criteria are met.

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

About Leber’s Hereditary Optic Neuropathy

Affecting approximately 35,000 people worldwide, LHON causes neuropathy of the optic nerve and retinal ganglion cells in the back of the eye and can lead to legal blindness. Mitochondrial dysfunction is a key factor in genetic optic neuropathies such as LHON, characterized by loss of visual function resulting from impaired cellular energetics. LHON primarily affects young men between the ages of 18 and 30. Vision problems, such as blurring or clouding of vision, may begin in one eye or both eyes at the same time. Over time, vision worsens in both eyes and often leads to severe and permanent legal blindness.

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 LHON – 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.

Source: Stealth BioTherapeutics

Posted: December 2017

Tagged with:

About author

Related Articles