Acen Pharmaceutical Bcl-2 inhibitors are re-qualified as FDA orphan drugs

Transfer from On September 7, Asathe Pharmaceuticals announced that the U.S. FDA had awarded it the qualification of APG-2575 orphan drug for the treatment of chronic lymphocytic leukemia (CLL), an innovative drug in the study.
this is the second FDA-granted orphan drug qualification for APG-2575.
, the drug was awarded the FDA's first orphan drug, WM.
APG-2575 is a new oral Bcl-2 selective small molecule inhibitor developed by Asaan Pharmaceuticals to restore the tumor cell program death mechanism (apoptosis) by selectively inhibiting Bcl-2 protein, thereby killing tumors, intended to be used to treat a variety of blood malignancies.
APG-2575 has been licensed for phase 1b/2 clinical trials in the United States, China and Australia and is advancing the clinical development of multiple blood tumor adaptations worldwide simultaneously.
A global Phase 1b/2 clinical study of relapsed/difficult-to-treat CLL/SLL (small lymphocyte lymphoma) is under way and is being recruited in the United States and Australia.
"orphan medicine", also known as rare drugs, refers to the prevention, treatment, diagnosis of rare diseases of drugs.
, rare diseases are those that affect fewer than 200,000 people.
since 1983, the United States has adopted the Orphan Drugs Act to provide policy support to businesses to encourage the development of drugs for rare diseases.
2020, the FDA granted APG-2575 orphan drug eligibility for treatment of Fahrenheit globulinemia.
this time, APG-2575 was awarded the second orphan drug by the FDA for the treatment of chronic lymphocytic leukemia.
According to the press release, this APG-2575 FDA-granted orphan drug eligibility will help the drug enjoy some policy support in the U.S. in terms of follow-up research and development and commercialization, including tax relief on clinical trial costs, exemption from NDA filing fees, access to research and development grants, and in particular, seven years of exclusive access to the U.S. market after approval.
CLL is an adult leukemia characterized by a large number of cloned B lymphocytes in lymphatic tissues such as perennity blood, bone marrow, spleen and lymph nodes.
2020, there will be about 21,040 new cases of CLL in the United States and about 4,060 deaths from the disease, according to the American Cancer Society.
, according to the latest SEER data, there will be fewer than 200,000 CLL patients in the U.S. by 2020.
The development of existing Bruton tyrosine protein kinase (BTK) inhibitors and Bcl-2 inhibitors has improved the prognostication of CLL patients, but there is still a medical need for safer and more effective treatment options that achieve deep remission in short-term treatments and are free of chemotherapy.
: Aceh Pharmaceutical Bcl-2 inhibitors have been re-qualified by the FDA for the treatment of chronic lymphocytic leukemia. Retrieved Sep 07, 2020, from Assin Pharma