9 Meters Biopharma Inc. (NASDAQ:NMTR) has received Orphan Drug designation from the US Food and Drug Administration. The grant is for its proprietary long-acting glucagon-like-peptide-2 (GLP-2) receptor agonist, NM-003 for preventing acute graft versus host disease (GvHD).
NM-003 receives Orphan Drug designation
The company has designed NM-003 which is also called teduglutide as a long-acting injectable GLP-2 receptor agonist utilizing the novel XTEN® tech to extend circulation half-life. Patrick Griffin, the company’s Chief Medical Officer said that they are evaluating the long-acting GLP-2’s applicability in preventing GvHD. GvHD is a problem for transplant recipients and it doesn’t have an effective pharmaceutical alternative for treatment or prevention. Griffins added that the company is also planning to advance to Investigational New Drug (IND) development stages from 2021 for the approved orphan indication. 9 Meters Biopharma could also consider other indications where it can bolster treatment setting for underserved patient populations.
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The FDA’s Office of Orphan Products Development (OOPD) usually grants orphan drug designation to facilitate the development and evaluation of effective and safe biologic and drugs that prevent, treat, or diagnose rare diseases. These are diseases that affect less than 200,000 people in the United States. According to the Orphan Drug Act, the designation qualifies biologics or drug sponsors for development perks conferred by the US FDA. These incentives include tax credits for approved clinical testing. Interestingly a marketing application for a drug that has received orphan designation will not be subject to prescription drug user fee except when the application covers another indication besides the rare condition or disease for which the orphan drug was designated.
GvHD results after allogeneic transplants
GvHD sometimes occur following allogeneic transplants such as hematopoietic stem cell or bone marrow transplants and sometimes after solid organ transplants. It develops when donor tissue white blood cells take the recipient tissue as non-self or foreign and thus respond by triggering an immune response which results in inflammation. Most importantly acute GvHD occurs within weeks to months after one has received a transplant. Sometimes severe cases can result in organ damage.