RARE Daily

FDA Grants Rare Pediatric Disease Designation to Retrotope’s Therapy for Two Neurodegenerative Indications

February 25, 2021

Rare Daily Staff

The U.S. Food and Drug Administration has granted two Rare Pediatric Disease designations to Retrotope’s lead therapy in development for the treatment of the life-threatening neurodevelopmental disorders infantile neuroaxonal dystrophy and Friedreich’s ataxia.

Infantile neuroaxonal dystrophy (INAD) 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 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.

Friedreich’s ataxia (FA) is a debilitating and life-shortening neurodegenerative disease that affects approximately 5,000 people in the United States and more than 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. 

“INAD and FA are both devastating diseases, not only for the patients affected but also for their loved ones,” said Vidhya Gopalakrishnan, chief development officer at Retrotope. “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.”

Retrotope’s lead candidate RT001 is a clinical-stage isotopically stabilized, synthetic linoleic acid (LA) discovered and developed with Retrotope’s platform technology, which 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 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. 

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The designation makes each RT001 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Rhythm Pharmaceutics sold its priority review voucher for $100 million in January 2021 to Alexion Pharmaceuticals.

RT001 has also been granted Fast Track designation by the FDA for the treatment of FA and orphan drug designation by the European Medicines Agency 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 and PLA2G6-associated neurodegeneration, which includes INAD.

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