Neurotechnology company Synapse Biomedical has received Humanitarian Device Exemption (HDE) marketing approval from the U.S. Food and Drug Administration for a diaphragm pacing system that helps certain types of patients suffering from amyotrophic lateral sclerosis with breathing.
The approval means that Oberlin, Ohio-based Synapse can begin marketing and selling the device for a subset of ALS patients.
“This is clearly a huge step for us,” said CEO Tony Ignagni.
The company’s NeuRx Diaphragm Pacing System electrically stimulates the diaphragm to contract, simulating a breathing motion, to help patients with inadequate breathing. The FDA approval applies to ALS patients who have stimulatable diaphragms and chronic difficulty in breathing, according to a statement from Synapse.
Repeated use of the device conditions the diaphragm muscles, delaying respiratory failure and the need for tracheostomy and mechanical ventilation. A 106-patient clinical trial of the device demonstrated that it could help ALS patients live longer and sleep better than the current standard of care, according to the statement.
Better known as Lou Gehrig’s disease, ALS is a disease of the nerve cells in the brain and spinal cord that control voluntary muscle movement. Around 6,000 U.S. patients per year are diagnosed with the disease, and about half of them would benefit from Synapse’s diaphragm pacing system, Ignagni said.
As for commercialization, Synapse plans to work closely with two groups that are heavily involved in the ALS community — the ALS Association and the Muscular Dystrophy Association. The groups have relationships with research centers that work with ALS patients, and those research centers and patients could become customers for Synapse, Ignagni said.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Launched in 2002, Synapse Biomedical commercialized technology developed largely by Dr. Ray Onders over two decades at Case Western Reserve University and University Hospitals Case Medical Center in Cleveland. The company has drawn attention for its association with the late actor Christopher Reeve, who became Synapse’s third patient after he was paralyzed due to a spinal cord injury in a fall from a horse.
“We were inundated with inquiries from the ALS community after that,” Ignagni said.
