In a recent press release, clinical stage biotech and biopharmaceutical company Biom Therapeutics (“Biom”) shared that its drug candidate BIO017 was granted Orphan Drug designation. The treatment, designed for patients with Angelman syndrome, could fulfill an unmet need in this patient population. Currently, there are no FDA-approved treatments for Angelman syndrome.

BIO017

Developed by Biom as the lead therapeutic candidate, BIO017 is described as a TRPV1 agonist. In addition to Angelman syndrome, BIO017 is being developed for patients with developmental and epileptic encephalopathies (DEEs). Preclinical studies highlighted BIO017’s abilities to prevent seizures and improve behavior in patients with Angelman syndrome.

Earlier in 2021, Biom submitted an Investigational New Drug (IND) application for BIO017. Now, the treatment has received Orphan Drug designation from the FDA. According to the FDA:

The Orphan Drug Act (ODA) provides for granting special status to a drug or biological product (“drug”) to treat a rare disease or condition upon request of a sponsor. This status is referred to as orphan designation (or sometimes “orphan status”).

To qualify for Orphan Drug status, a drug or biologic must treat a “rare” condition, defined as those which affect under 200,000 Americans. Once a drug receives this designation, the sponsor (in this case, Biom) is eligible for up to 7 years of market exclusivity, additional FDA assistance, tax credits, and fee waivers. Ultimately, by the end of the year, Biom hopes to continue evaluating BIO017 for patients with Angelman syndrome through the initiation of clinical trials.

Angelman Syndrome

Typically, Angelman syndrome, a rare neurological and genetic disorder which affects the nervous system, is caused by chromosome 15 defects. Patients either have a missing or defective gene. However, because most patients with Angelman syndrome have no family history of the condition, doctors are not exactly sure what causes this genetic malfunction. In many cases, Angelman syndrome is misdiagnosed as either autism or cerebral palsy. Despite a lack of cures or treatments, patients with Angelman syndrome will often live for a normal lifespan. Signs and symptoms include:

• Developmental and learning delays
  • Note: For example, an infant who does not crawl or babble may be displaying developmental delays.
• Happy, excitable personalities
  • Note: At some points, patients may display laughing, smiling, or talking at inappropriate times.
• Speech impairment, such as minimal or no speech capabilities
• Small head size
• Seizures
• Difficulty walking or moving
• Tongue thrusting or hand-flapping
• Difficulty sleeping or other sleep disorders
• Stiff or jerky movements
• Feeding difficulties
• Obesity

Learn more about Angelman syndrome.