Longeveron Inc (NASDAQ:LGVN) announced the US FDA’s approval to use its Investigational Cell Therapy – Lomecel-B to treat children suffering from HLHS ( Hypoplastic Left Heart Syndrome).
Longeveron manufactures Lomecel-B, an allogeneic, bone (marrow-derived medicinal signaling cell) MSC, under cGMP at its cell processing facility based in Miami, Florida. The drug will be administered by the Division Head (Cardiovascular Thoracic Surgery), Sunjay Kaushal, at Ann & Robert H. Lurie Children’s Hospital.
Patients gains access to IND
The patients can gain access to investigational drugs, medical devices, and biologics under an expanded access program permitted by the US FDA. Physicians can monitor, diagnose, or cure the patients who suffer from serious conditions or diseases, using IND where no satisfactory therapy options are available. The protocol is reviewed and approved by the internal review board (IRB) of Lurie Children’s Hospital.
In the US, Dr. Kaushal is the first surgeon for administering the MSC to a small baby suffering from HLHS. Kaushal said its main target is to improve right ventricle functioning by regenerating cardiac tissue and act similar to a left ventricle in pumping.
Kaushal expressed his gratitude to IRB of Lurie Children’s Hospital, the FDA, and Longeveron for making this miracle happen. He hopes this treatment will be a game-changer for the babies.
CEO of Longeveron, Geoff Green, said its main aim is to offer an innovative treatment option for babies with HLHS using the previous studies. Lomecel-B’s MSCs expect to improve vascular and ventricular functioning. The rare congenital heart defect – HLHS affects annually 1,000 babies in the US.
Some babies’ heart loses its ability to pump sufficient blood to the body because of the underdeveloped left ventricle. It requires a TRS (three-staged reconstructive surgery), which is often dangerous. Even after surgery, the baby may experience cardiac failure and requires heart transplantation.
Longeveron recently completed Phase 1 clinical trial of Lomecel-B in children with HLHS. The study is lead by renowned pediatric cardiac surgeons from the University of Cincinnati, the University of Maryland Medical Center, and the University of Utah Primary Children’s Hospital. The company received financial support from Maryland Stem Cell Research Fund.
No adverse side effects are observed in the clinical trial of intramyocardial injection of Lomecel-B. The babies well tolerate it. Longeveron will conduct the Phase 2 clinical study for advancing its HLHS program in Q3 2021.