RARE Daily

FDA Grants Rare Pediatric Disease Designation to Therapies from Moleculin and Akuous

April 14, 2021


Rare Daily Staff    

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Moleculin’s experimental drug WP1066 for the treatment of ependymoma, and both Orphan Drug and Rare Pediatric Disease designation to Akuous’ experimental gene therapy AK-OTOF for the treatment of otoferlin gene-mediated hearing loss.

Ependymoma is a rare type of tumor that can form in the brain or spinal cord. Ependymoma begins in the ependymal cells in the brain and spinal cord that line the passageways where the fluid (cerebrospinal fluid) that nourishes your brain flows. Ependymoma can occur at any age, but most often occurs in young children.

Moleculin’s WP1066 is an immune/transcription modulator capable of directly inhibiting certain key oncogenic transcription factors, including the activated form of a protein known as STAT3. The activated form of STAT3 referred to as p-STAT3, is considered a master regulator of tumor activity. In addition to inhibiting p-STAT3 and several other signaling proteins linked to tumor development, WP1066 has also been shown in animal models to stimulate a natural immune response and immune memory to fight tumor progression.

“The FDA’s recognition of the high prevalence and unmet needs in the treatment landscape for ependymoma, especially in pediatric patients is a significant milestone as we continue to advance and expand the WP1066 development program. We currently have Orphan Drug Designation for WP1066 for the treatment of brain tumors, as well as RPD designation for three other pediatric indications, and believe that ependymoma represents another important rare indication,” said Walter Klemp, chairman and CEO of Moleculin.

Otoferlin gene (OTOF)-mediated hearing loss is a form of sensorineural hearing loss caused by mutations in the OTOF gene. The OTOF gene encodes otoferlin, a protein that enables the inner hair cells of the cochlea to release neurotransmitter vesicles in response to stimulation by sound to activate auditory neurons. Most individuals with OTOF-mediated hearing loss have severe-to-profound sensorineural hearing loss from birth and approximately 20,000 individuals are affected in the United States and Europe. AK-OTOF is designed to treat the underlying cause of OTOF-mediated hearing loss through delivery of a transgene using a dual vector technology that results in expression of normal, functional otoferlin protein in the affected cells, namely inner hair cells, in the cochlea.

“There are currently no pharmacologic treatment options for individuals with OTOF-mediated hearing loss, or for any other form of sensorineural hearing loss. The nonclinical data reported to date for AK-OTOF demonstrate durable recovery of auditory function and support future clinical development.” said Jen Wellman, chief operating officer of Akouos. “We believe these are the first Orphan Drug and Rare Pediatric Disease designations granted by FDA for a genetic form of hearing loss, and they represent an important milestone for the field of inner ear genetic medicines.”

Akouos anticipates that it will submit an investigational new drug application (IND) for AK-OTOF in the first half of 2022.

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 Moleculin’s WP 1066 and Akuous’ AK-OTOF 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.

The FDA grants Orphan Drug designation to drugs and biologics intended for the treatment, diagnosis, or prevention of rare diseases, or conditions affecting fewer than 200,000 people in the United States. The designation affords the potential for certain benefits, including up to seven years of post-approval market exclusivity, assistance in the drug development process, tax credits for clinical development, and exemptions from certain FDA fees.

Photo: Jen Wellman, chief operating officer of Akouos


Stay Connected

Sign up for updates straight to your inbox.