Retrotope Granted Rare Pediatric Disease Designation from FDA for Lead Development Candidate, RT001, in Two Life-Threatening Neurodegenerative Indications

Los Altos, California, UNITED STATES

Company Currently Conducting Late-Stage Clinical Trials in Both Infantile Neuroaxonal Dystrophy (INAD) and Friedreich’s Ataxia (FA)

RT001 also Granted Fast Track Designation by FDA in FA; Orphan Drug Designation by European Medicines Agency in INAD

LOS ALTOS, Calif., Feb. 25, 2021 (GLOBE NEWSWIRE) -- Retrotope, a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted two rare pediatric disease designations to RT001, the company’s lead development candidate. The first rare pediatric disease designation is for the treatment of infantile neuroaxonal dystrophy (INAD), with the second covering the treatment of Friedreich’s ataxia (FA). In addition, RT001 has been granted Fast Track designation by the FDA for the treatment of FA and orphan drug designation by the European Medicines Agency (EMA) for the treatment of INAD.   RT001 has previously been granted orphan drug designation in the U.S. for the treatment of multiple diseases, including FA, progressive supranuclear palsy (PSP) and PLA2G6-associated neurodegeneration, which includes INAD.

Rare pediatric disease designation is granted by the FDA to drug candidates being developed to address serious and life-threatening diseases that primarily affect children, with onset of severe symptoms occurring prior to 18 years of age. To qualify for rare pediatric disease designation, these diseases must affect fewer than 200,000 people in the United States.   Under the FDA's rare pediatric disease priority review voucher program, a sponsor that receives an approval for a drug that has been granted rare pediatric disease designation may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product or sold to other companies. Retrotope is eligible to receive a priority review voucher should the company secure approval for RT001 in either one of these indications.

The FDA’s Fast Track program is designed to facilitate the development, and expedite the review of, medicines to treat serious or life-threatening conditions and fill an unmet medical need. Recipients of Fast Track designation are eligible for greater access to the FDA during the clinical development process and may also qualify for priority review. 

Orphan drug designation may be granted by the EMA to therapeutic candidates that are intended to treat a serious condition that affects fewer than five in 10,000 people in the EU, and which demonstrate sufficient data to suggest the candidate may produce clinically relevant outcomes. The designation provides companies with certain benefits and incentives for clinical development and a period of market exclusivity, if approved.

“We are proud to receive these key regulatory designations for RT001 in INAD and FA, as they validate and support the important work that we are undertaking to address rare, life-threatening diseases that are impacting young people around the world,” said Vidhya Gopalakrishnan, Ph.D., Retrotope’s chief development officer. “INAD and FA are both devastating diseases, not only for the patients affected but also for their loved ones. At Retrotope, we are working diligently to advance RT001 through late-stage clinical trials in both INAD and FA and remain committed to the goal of delivering a treatment to these patients that can make a meaningful difference in their lives.”

RT001 is a clinical-stage isotopically stabilized, synthetic linoleic acid (LA) discovered and developed with Retrotope’s novel platform technology. This platform is designed to combat the oxidative stress and cellular degeneration that arises from lipid peroxidation (LPO). While all healthy human tissues undergo this physiological process of cell degeneration and repair, it is well-established that a wide range of serious degenerative diseases are precipitated when the LPO process gets out of balance. Polyunsaturated fatty acids (PUFAs), which make up cell and mitochondrial membranes throughout the body and are vital to healthy cellular function, are the target of the LPO process due to their inherent instability. Free radicals in the body exploit the instability of PUFAs to trigger chain reactions that drive LPO and the resulting degradation of these vital PUFAs. Retrotope’s novel platform technology creates stabilized PUFAs, such as RT001, that become an integral part of all membranes and are capable of down-regulating LPO in order to protect membranes from degeneration. RT001 has been safely administered orally on a daily basis to more than 100 patients, spanning more than 1,000 patient months.

Retrotope is currently conducting a potentially pivotal Phase 2/3 trial of RT001 in patients with INAD. The company has completed dosing and expects data to read out from the study in the first half of 2021. Additionally, the company is currently conducting a pivotal Phase 2/3 trial of RT001 in patients with FA. Enrollment in this study was completed in late 2020 and data is expected to read out by the end of 2021.  

“These designations awarded to Retrotope are aligned with the high unmet need and call for urgency in the FA community. FA most commonly has onset of symptoms during childhood, ages 10-15 years, with significant progression and functional loss taking place during this time. We believe it is important to conduct clinical trials in both children and adults with FA as intervening early will likely have the biggest therapeutic benefit in the long term,” said Jennifer Farmer, chief executive officer, Friedreich’s Ataxia Research Alliance (FARA). “The entire team at FARA is grateful for the dedication that Retrotope has demonstrated in its effort to develop a novel treatment for FA. We are excited that the company has completely enrolled its ongoing pivotal study of RT001 in patients with FA and look forward to the read out of data from the trial.”

About Infantile Neuroaxonal Dystrophy (INAD)

Infantile Neuroaxonal Dystrophy is an ultra-rare, infantile genetic neurological disorder and part of a spectrum of diseases called PLA2G6-associated neurodegeneration. Symptoms usually present between six and 18 months of age and there is often rapid onset of motor and intellectual regression. Later on, diminished muscle tone (hypotonia) and spasticity develop. The disease also leads to problems with vision and the eyes, the autonomic nervous system, and, in a minority of individuals, seizures. Usually, disease progression is rapid, and the disorder is invariably fatal in childhood.   There are currently no approved treatments for INAD.

About Friedreich’s Ataxia (FA)

Friedreich’s Ataxia is a debilitating and life-shortening neurodegenerative disease that affects approximately 5,000 people in the United States and over 20,000 people worldwide. FA results in the progressive loss of coordination and muscle strength which then leads to motor incapacitation, wheelchair dependence, and ultimately early death most commonly due to cardiomyopathy. There are currently no approved treatments for FA.  

About Retrotope

Retrotope is a clinical-stage biopharmaceutical company focused on the development of first-in-class therapies for degenerative diseases ranging from orphan neurodegenerative indications to large market degenerative conditions. The company leverages its proprietary drug discovery platform to create novel, disease-modifying drugs designed to combat the oxidative stress and cellular degeneration that arises from lipid peroxidation (LPO). It does so through the creation of isotopically stabilized synthetic versions of polyunsaturated fatty acids (PUFAs) that trigger the downregulation of the LPO process. The company’s lead development candidate, RT001, is a clinical-stage isotopically stabilized, synthetic linoleic acid (LA) that is in development for a range of orphan neurodegenerative diseases including infantile neuroaxonal dystrophy (INAD), Friedreich’s ataxia (FA), amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and progressive supranuclear palsy (PSP). In addition, the company is advancing its second development candidate, RT011, an isotopically stabilized, synthetic docosahexaenoic acid (DHA), toward the clinic for the treatment of dry age-related macular degeneration (AMD).

For more information, please visit

Vida Strategic Partners 
Stephanie Diaz (Investors)Tim Brons (Media)