In the past week, the US FDA has issued 15 orphan drug qualifications in one breath, including many drugs for treating liver cancer, gastric cancer, and biliary tract cancer.
Of these 15 drugs, 5 are used for cancer treatment, accounting for one third. Among them, the cell therapy TCB002 brought by TC BioPharm is the only drug for blood cancer treatment. This is an "allogeneic" "ready-to-use" cell therapy for expanded CD3-positive gamma delta T cells. It has entered clinical trials to treat acute myeloid leukemia.
The remaining four cancer therapies treat solid tumors, of which AstraZeneca's PD-L1 inhibitor Imfinzi (durvalumab) and CTLA-4 inhibitor tremelimumab are orphan drug qualifications for hepatocellular carcinoma. Silydi's subcutaneous PD-L1 antibody envafolimab is eligible for orphan drug targeting biliary tract cancer. OBI-999 of Taiwan Hao Ding Company is an antibody-conjugated drug against the Globo-H antigen, and its orphan drug qualification is targeted at gastric cancer.
We have also seen some gene therapies, such as one from Esteve Pharmaceuticals, which aims to use the AAV9 gene vector to introduce the normal GALNS gene into patients to treat IVA mucopolysaccharidosis; another treatment by Baxalta is The gene encoding FIX Padua was transferred into patients with AAV8 vector to treat hemophilia type B.
Aruvant Sciences is a therapy using lentiviral vectors. It introduces a gene encoding γ-globin into a patient's own CD34-positive cells to treat sickle cell anemia. Another similar therapy comes from Orchard Therapeutics, which also uses CD34-positive cells. The difference is that it transfers the gp91phox gene to treat X-chromosome-linked chronic granulomatosis.
Other rare diseases
In addition to cancer and rare diseases that can be treated with gene therapy, other rare diseases also have potential new drug developments. Woolsey Pharmaceuticals 'fasudil is a ROCK inhibitor, which is expected to treat amyotrophic lateral sclerosis; OncoArendi Therapeutics' OATD-01 is an innovative CHIT-1 inhibitor, which is expected to be used for the treatment of sarcoidosis; Rebasez (Luspatercept) is a red blood cell maturation agent, and the disease targeted by orphan drug qualification is bone marrow fibrosis.
Among these drugs that qualify for orphan drugs, there are also some anti-infective drugs. Among them, Danuo Medicine's TNP 2092 obtained the qualification for artificial joint infection. Previously, it has also been qualified by the FDA as an anti-infective product. F2G's olorofim has obtained two orphan drug qualifications, respectively for the infection of Lomentospora / Scedosporium and invasive aspergillosis.