Moleculin Biotech Inc (NASDAQ:MBRX), a major biotech stocks has announced that Annamycin also referred to as Liposomal Annamycin, the company’s leukemia drug candidate has been granted Orphan Drug status by the Food And Drug Administration (FDA) . The drug is used in the treatment of acute myeloid leukemia (AML).
The status which is highly coveted, comes with several incentives to makers of drugs such as expedited approval by FDA, removal of restrictions on fixing prices of the drug used in the treatment of any of the 7,000 diseases, the drug maker gets seven years of exclusive access to the marketplace even when the patent expires. The drug maker gets a 50% R&D tax credit and a waiver of the fee charged for the New Drug Application (NDA).
Although the status of orphan drug is beneficial in the development of much needed treatment for many rare diseases, a new report has showed that drug makers often misused the incentives offered under the scheme for monetary gains.
Acute myeloid leukemia (AML) is a cancer of the blood that involves abnormal cell division and growth. The bone marrows are filled with immature blasts of white blood cells. The Acute myeloid leukemia spreads rapidly and the cancer cells affect the production and development of normal red blood cells, white blood cells and the platelets. AML is mainly treated through bone marrow transplant and the patients must qualify for the transplant by going through an induction therapy which is done using an antibiotic called anthracyclines.
Annamycin is a type of anthracycline that has been specifically designed to wash out cardio-toxicity which can lead to heart problems. The drug also performs better than the current anthracyclines by preventing the multi-drug resistance mechanisms. During the early stages of the study, the drug showed high levels of efficacy by clearing leukemic blasts completely.
Other companies and top biotech stocks which are working on drugs for various indications of AML are Actinium Pharmaceuticals Inc (NYSEMKT:ATNM) which is currently working on Iomab-B that was granted an EMA recently.
Moleculin Biotech is based in Houston, Texas. The pharmaceutical company has several other drugs that are still at various stages of clinical studies.