▎WuXi AppTec/Report
Source: Pixabay
After 10 drugs obtained " orphan drug qualification" last week, the number of "orphan drug qualifications" this week reached 10 again. In today's article, the WuXi AppTec content team will provide background information on these 10 new drugs.
Drug Name: BF844 Research and Development Company: Usher III Initiative Treatment Disease: Usher Syndrome Drug Description: Usher syndrome is a genetic disease that causes progressive deterioration of vision and hearing in patients. Among them, type III Usher syndrome is the rarest type, due to mutations in the CLRN1 gene and the HARS gene. In drug screening, the researchers found small-molecule drugs that could stabilize the mutant CLRN1 protein. In mouse models, it effectively alleviates the problem of hearing loss. Human clinical trials are expected to begin in the next few years.
Drug Name: FN-1501 R&D Company: Fosun Pharma Treatment Disease: Acute Myeloid Leukemia Drug Introduction: FN-1501 is an inhibitor that can target to multiple tyrosine kinases. A Journal of Clinical Oncology study showed that it can target CDK4/6, PDGFR, KIT, ALK, RET, and FLT3 proteins. Currently, it is in Phase 1 clinical trials.
Drug name: dalfampridine R&D company: Magnum Therapeutics Treatment disease: Transverse Myelitis Drug introduction: Dalfampridine is a potassium channel inhibitor, which is expected to promote the signal transduction of nerve fibers. In 2010, it was approved by the US FDA to improve the walking ability of patients with multiple sclerosis.
Drug Name: Δ9-Tetrahydrocannabinol R&D Company: Tetra Bio-Pharma Treatment Disease: Hepatocellular Carcinoma Drug Introduction: Tetra Bio-Pharma is a company focused on the development of cannabinoid-derived drugs.On the FDA's official website, this orphan drug designation targets "Treatment of Hepatocellular Carcinoma". But according to the company's official website, the drug is more likely to treat pain caused by tumors.
Drug name: Hypotonic acid oxidizing solution containing hypochlorous acid Research and development company: APR Applied Pharma Research Treatment disease: Epidermolysis bullosa One of the most painful diseases". Due to the lack of certain key proteins in the skin, patients experience painful blisters on the skin after light touch. The company's proprietary nanotechnology facilitates the production of hypochlorous acid solutions to help patients' wounds heal.
Drug Name: nicardipine R&D Company: Collaborations Pharmaceuticals Treatment Disease: Pitt-Hopkins Syndrome Drug Description: Pitt-Hopkins Syndrome is a genetic disorder that causes developmental delays in patients And a series of problems, affecting intelligence and athletic ability. From the perspective of etiology, the mutation of TCF4 transcription factor accounts for a large proportion, which will increase the levels of Kv7.1 and Nav1.8. Therefore, with inhibitors of these two ion channels, it is expected to control the symptoms of Pitt-Hopkins syndrome. As a calcium channel antagonist, nicardipine is expected to achieve similar effects.
Drug Name: Gene Therapy Expressing Human CLN3 Gene R&D Company: Amicus Therapeutics Treatment Disease: CLN3-mutated Batten Disease Drug Introduction: Batten Disease is also known as neuronal ceroid lipofuscinosis ( neuronal ceroid lipofuscinosis, NCL), caused by mutations in a series of CLN3 genes. The therapy introduces the normal CLN3 gene into the patient through the AAV vector to relieve the patient's condition.
Drug Name: Gene Therapy Expressing Human CLN6 Gene R&D Company: Amicus Therapeutics Treatment Disease: CLN6 Mutated Batten Disease Drug Introduction: The introduction is the same as above. The difference is that this therapy targets Barton's disease with CLN6 mutations.
Drug Name: APVO436 R&D Company: Aptevo Research and Development Treatment Disease: Acute Myeloid Leukemia Drug Introduction: APVO436 is a bispecific antibody against CD3 and CD123, which has entered the Phase 1 clinical stage. Early data in July showed it was well tolerated and no antibody production against the drug was observed.
Drug Name: romiplostim R&D Company: Amgen (Amgen) Treatment Disease: Aplastic Anemia Drug Introduction: Romiplostim is a thrombopoietin receptor agonist, which can simulate the human body's natural thrombopoietin effect, thereby Raise platelet counts. It has previously received multiple FDA approvals for the treatment of immune thrombocytopenia and chronic immune thrombocytopenic purpura.
Orphan Drug Weekly Review:
November 9, 2019: 4 drugs on the list, 2 from China, for the treatment of cholangiocarcinoma and esophageal cancer
November 16, 2019: Amgen bispecific antibody on the list, Axovant gene therapy finalist November 23, 2019: Boom! This week, 10 new drugs have been granted orphan drug status, and both Hutchison and BeiGene have been selected as
Copyright note: This article is from WuXi AppTec content team. Individuals are welcome to forward it to the circle of friends. Unauthorized use of any media or institutions is prohibited. The form is reproduced to other platforms.
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-