PTC Therapeutics, Inc. (NASDAQ: PTCT) has completed meetings with CAT (Committee for Advanced Therapies). The meeting focused on the company using its gene therapy to treat AADC deficiency. CAT usually assesses advanced therapies and their efficacy and safety. If the body approves the therapy, AADC will be the only gene therapy that is brain infused.
According to PTC Therapeutics’ CEO, Stuart W. Peltz, the company hopes to soon treat patients with AADC deficiency using its therapy. The company believes the drug could significantly improve the lives of people with the disease.
The company carried out there successful clinical trials
PTC Therapeutics’ decision to market the gene therapy stems from the success of 3 clinical trials it did in Taiwan. The company considered this data as it submitted its application for marketing authorisation.
It also considered its efficacy on two patients who were part of a compassionate treatment move. The company administered the drug via stereotactic surgery. This procedure is minimally invasive. Physicians use it to treat neurological disorders in children and adults.
AADC deficiency is an inherited genetic disorder. The disease is fatal and results in severe disabilities. It affects patients behaviorally, mentally, and physically from infants. Children with the disease could have daily seizures that resemble oculogyric crises.
The seizures, which could occur for hours, can occur daily. They are associated with eye-rolling, gastrointestinal tract issues, respiratory infections, sleeping difficulties, behavioural problems, and frequent vomiting.
Complications that result from AADC deficiency
No regulator has approved disease-modifying medication for AADS deficiency. Moreover, children with the disorder often require several medications to manage their symptoms. They also require supportive therapy, including speech, occupation, and physical therapy.
In some cases, patients could require surgery if they have severe complications like breathing and feeding issues. The disease is also rarely diagnosed despite there being many tests for it.
PTC Therapeutics is a biotechnology company that discovers, develops, and commercialises therapies for people with rare diseases. The company’s goal is to help patients with diseases that have high unmet medical needs.
PTC uses its innovative and experienced team to achieve its goals. It has also achieved success in commercialising its products globally, thus increasing value for its shareholders.
