Breaking News
February 17, 2018 - ‘A Time Clock to a Tissue Clock’ for Acute Stroke Care
February 17, 2018 - Cancer Care Gets Personal | NIH News in Health
February 17, 2018 - Do more youth use or do youth use more?
February 17, 2018 - Eating faster linked to obesity
February 17, 2018 - Who’s Still Smoking? ACS Report Highlights Most Vulnerable Adults
February 17, 2018 - Study of smoking and genetics illuminates complexities of blood pressure
February 17, 2018 - Study reveals new link between bone cells and blood glucose level
February 17, 2018 - Children with reading challenges may have lower than expected binocular vision test results
February 17, 2018 - Mass Shootings Trigger Change for Emergency Medicine
February 17, 2018 - ECMO helps revive woman thought to be drowned
February 17, 2018 - Learning stress-reducing techniques may benefit people with epilepsy
February 17, 2018 - Shedding Pounds Before Weight-Loss Surgery a Smart Move
February 17, 2018 - FDA Approves New Cystic Fibrosis Drug Combo
February 17, 2018 - Augmented Reality helps surgeons to ‘see through’ tissue and reconnect blood vessels
February 17, 2018 - Emotional state affects operation of the entire brain instead of being restricted to specific regions
February 17, 2018 - Apalutamide Slows Metastasis in Prostate Cancer
February 17, 2018 - Kids’ well visits linked to lower appendicitis complications
February 17, 2018 - New NK cell-based immunotherapy effective against several types of leukemia
February 17, 2018 - Producing Super-Swelled Lyotropic Crystals for Drug Development
February 17, 2018 - Pfizer Receives Breakthrough Therapy Designation from FDA for PF-04965842, an oral JAK1 Inhibitor, for the Treatment of Patients with Moderate-to-Severe Atopic Dermatitis
February 17, 2018 - Molecular Imaging Flags Risk of AAA Rupture
February 17, 2018 - Researchers identify risk factors for sleep apnea during pregnancy
February 17, 2018 - More work required to find the right drug dosage for pediatric patients
February 17, 2018 - Factors ID’d That Predict RA Remission with Etanercept
February 17, 2018 - A handout or a hand up? How we judge others guides how we help others
February 17, 2018 - ACR receives grant to focus on projects that reduce health disparities
February 17, 2018 - Pimavanserin Might Be Safer Alternative to Ease Dementia Psychosis
February 17, 2018 - Risks of Lung Screening Seen Outweighing Benefits in Many with Smoking History
February 17, 2018 - The impact of Hurricane Harvey on pregnant moms
February 17, 2018 - Gene editing tool used to detect cancer
February 17, 2018 - Researchers detail molecular atlas of cells that form brain’s blood vessels
February 17, 2018 - TUM scientists observe formation of myelin sheaths around nerve fibers
February 17, 2018 - Worst Flu Season Yet? | Medpage Today
February 17, 2018 - Finding the root cause of bronchiolitis symptoms
February 17, 2018 - Climbing stairs reduces hypertension and strengthens muscles
February 17, 2018 - Nature paper unveils bacterial division
February 16, 2018 - Postoperative pain control following extensive pelvic exenteration
February 16, 2018 - Daré Bioscience, Inc. Enters into License and Collaboration Agreement for a Product with the Potential to Receive the First FDA Approval for Female Sexual Arousal Disorder
February 16, 2018 - Havana Embassy Staff: ‘Concussion Without Concussion’?
February 16, 2018 - Family impact of congenital Zika syndrome likely to last a lifetime
February 16, 2018 - STI Prevention Helped By Also Discussing Pot, Alcohol Use
February 16, 2018 - New method maps the dopamine system in Parkinson’s patients
February 16, 2018 - Monitoring the Environment of Aseptic Processes
February 16, 2018 - Study finds decline in number of clinical trials funded by NIH
February 16, 2018 - Scientists show connection between sugar chains and bone growth
February 16, 2018 - Researchers develop new method for producing personalized medicine
February 16, 2018 - Women exposed to cleaning products suffer decreased lung function, Study finds
February 16, 2018 - Stem cell vaccine helps protect mice against numerous cancers
February 16, 2018 - Does Your Valentine Have a Roving Eye? Watch Out
February 16, 2018 - All Your MIPS Questions Answered — Sort Of
February 16, 2018 - Mitochondria may protect brain against Parkinson’s
February 16, 2018 - Key proteins could help in controlling the risk of osteoarthritis during aging, Study finds
February 16, 2018 - New review examines effectiveness of cupping therapy in athletes
February 16, 2018 - Resolving Interfacial Protein Dynamics by STReM
February 16, 2018 - Study provides new insights on why healthy children die from flu
February 16, 2018 - Self-sampling followed by HPV testing can benefit women at risk of cervical cancer
February 16, 2018 - Biomedical engineers grow living windpipe structures from self-assembled modules
February 16, 2018 - New device could allow people with disabilities to live more independently, enhance their quality of life
February 16, 2018 - Research identifies gene variants that play key role in how ovarian cancer patients process chemotherapy
February 16, 2018 - iMedicalApps: OB Wheels App Review
February 16, 2018 - To improve self-control, call weight loss what it is: Difficult
February 16, 2018 - Cervical tumors may be vulnerable to therapies that attack cancer’s fuel supply, study shows
February 16, 2018 - Allergan Announces Positive Top Line Phase 3 Results for Ubrogepant – an Oral CGRP Receptor Antagonist for the Acute Treatment of Migraine
February 16, 2018 - Morning Break: Hello Not-So-Fresh; One Eye, 14 Worms; Foreign Accent Syndrome
February 16, 2018 - New medical advances marking the end of a long reign for ‘diet wizards’
February 16, 2018 - Researchers study how unexpected event makes people to stop an action
February 16, 2018 - Women using short-acting asthma relievers take longer to become pregnant, study shows
February 16, 2018 - Fathers maybe passing on ovarian cancer genes to their daughters
February 16, 2018 - Phonak expands its latest-generation Belong platform with Phonak Naída B, Phonak Sky B hearing aids
February 16, 2018 - FDA Approves Erleada (apalutamide) for Non-Metastatic Castration-Resistant Prostate Cancer
February 16, 2018 - Traces of caffeine and its byproducts in the blood can be indicative of Parkinson’s disease
February 16, 2018 - More Help Needed With Opioid Crisis, Senators Told
February 16, 2018 - Are women really under-represented in clinical trials?
February 16, 2018 - US-based clinical study highlights safety and effectiveness of MENTOR MemoryShape Gel Breast Implants
February 16, 2018 - Higher levels of lifestyle physical activity linked to more gray matter in older adults’ brains
February 16, 2018 - Pfizer to use BC Platforms’ technology solutions to analyze data in cardiovascular diseases
February 16, 2018 - Researchers find shortcomings in pregnancy and prenatal care for women with diabetes
February 16, 2018 - Study could lead to new therapies to improve movement control in stroke survivors
February 16, 2018 - Do Common Household Chemicals Affect Your Weight?
February 16, 2018 - Binocular Video Game Tx Disappoints in ‘Lazy Eye’ Trial
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