The FDA approved five new drugs in November 2022, including two new molecular entities approved by the Center for Drug Review and Research (CDER), a combination therapy and an improved drug, and a gene therapy approved by the Center for Biologics Review and Research (CBER).
ELAHERE
ELAHERE (mirvetuximab soravtansine-gynx) is a first-in-class ADC developed by ImmunoGen that targets FRα, a cell surface protein highly expressed in ovarian cancer. Accelerated FDA approval for the treatment of folate receptor alpha (FRα) -positive, platinum-resistant epithelial ovarian cancer, fallopian tube cancer, or primary peritoneal cancer in adult patients who have previously received one to three systemic therapy regimen.Dr.
This is the first FDA-approved ADC for a platinum-resistant disease, and the approval is based on results from the pivotal Phase 3 trial SORAYA. Results: Among 106 patients with platinum-resistant ovarian cancer whose tumors expressed high levels of FRα, the objective response rate (ORR) was 31.7% (95%CI: 22.9%, 41.6%), including 5 patients with complete response (CR). The median DOR was 6.9 months (95%CI:5.6, 9.7).
In China, in 2020, East China Pharma will acquire exclusive clinical development and commercialization rights for the drug in mainland China, Hong Kong, Macao and Taiwan with an initial payment of US $40 million and a milestone payment of up to US $265 million.
TZIELD
TZIELD (teplizumab-mzwv) is an anti-CD3 monoclonal antibody developed by Provention Bio for intravenous administration that is approved to delay the course of disease in certain high-risk populations of type 1 diabetes.
It is the first and only approved drug to delay the onset of type 1 diabetes, fundamentally altering the course of the disease rather than just treating its symptoms. The FDA's approval was based on a clinical trial called TN-10. In this study, 76 patients with stage 2 type 1 diabetes were evaluated for the safety and efficacy of Tzield. The results showed that at a median follow-up of 51 months, 45 percent of the 44 patients who received Tzield were later diagnosed with stage 3 type 1 diabetes, compared to 72 percent of the 32 patients who received placebo. In addition, the median time from randomization to diagnosis of stage 3 type 1 diabetes was 50 months for patients receiving Tzield and 25 months for patients receiving placebo. These data suggest that TZIELD's treatment could significantly delay the progression of type 1 diabetes.
IMJUDO
Imjudo (tremelimumab) is an anti-CTLA-4 antibody developed by Astrazeneca, and Imfinzi (durvalumab) is an anti-PD-L1 monoclonal antibody.
Last month, the FDA approved Imjudo+Imfinzi for use in unresectable hepatocellular carcinoma. The FDA reapproved Imjudo+Imfinzi+ chemotherapy for adult patients with metastatic non-small cell lung cancer (NSCLC) who do not carry epidermal growth factor receptor (EGFR) mutations or allotrophic lymphoma kinase (ALK) fuses.
SEZABY
SEZABY (phenobarbital Sodium Powder for injection) is a new preparation of phenobarbital sodium developed by Sun Pharmaceutical. It is a benzyl alcohol-free and propylene glycol phenobarbital sodium powder. The drug was granted orphan drug status by the US FDA for the treatment of neonatal epilepsy.
This approval makes the drug the first and only FDA approved product specifically for the treatment of neonatal seizures.
HEMGENIX
HEMGENIX(etranacogene dezaparrvovec drlb) is a one-time gene therapy developed by uniQure and CSL for the treatment of hemophilia B in adults currently treated with clotting factor IX prophylactically or with current or historical life-threatening bleeding. Or have repeated severe spontaneous bleeding attacks. It is the world's first approved gene therapy for hemophilia B.
HEMGENIX uses the AAV5 vector to deliver a transgene expressing the Padua variant of clotting factor IX to the liver, causing the body to continuously produce factor IX and reducing abnormal bleeding rates in eligible hemophilia B patients. The treatment has previously been granted breakthrough therapy designation, orphan drug status and priority review status by the US Food and Drug Administration. Remarkably, the drug is priced at $3.5 million, making it one of the most expensive drugs available in the United States.
In November 2022, China's National Medical Products Administration (NMPA) approved three new Class 1 drugs, all of which are small molecule chemicals.
Toludivenlafaxine hydrochloride sustained release tablets
Torudivenlafaxine hydrochloride (Ansufaxine hydrochloride sustained release tablets, LY03005) was approved by the State Food and Drug Administration for the treatment of depression. This is China's first class 1 chemical medicine with independent research and development and independent intellectual property rights for the treatment of depression.
This drug was developed by Shandong Luye Pharmaceutical. Public data showed that the antidepressant effect of this drug may be related to the enhancement of 5-HT and NE effects in the central nervous system by inhibiting the reuptake of 5-HT and norepinephrine (NE).
Limplese tablets
linperlisib was conditionally approved by the National Food and Drug Administration for marketing in adults with relapsed or refractory follicular lymphoma who have received at least two prior systemic therapies. This is the first highly selective PI3Kδ inhibitor approved for market in China.
The drug was developed by Maya Li Pharmaceutical Shanghai. In February 2021, Hengrui made an equity investment of US $20 million (accounting for 6.67% of the total share capital) in Maya Li Pharmaceutical, and obtained the joint development rights and exclusive commercialization rights of Lin Plese in Greater China.
Limplitzer has previously received breakthrough therapy designation from the NMPA for the treatment of R/R FL and three orphan drug designations from the U.S. Food and Drug Administration for FL, chronic lymphocytic leukemia/small lymphocytic Lymphoma (CLL/SLL) and T-cell lymphoma indications. The most recent data from a Phase II clinical trial of Limprixel in R/R FL patients in China, published in 2022, showed an effective response rate of nearly 80% in patients with R/R FL and demonstrated a manageable safety profile.
Toluenesulfonamide injection
Toluenesulfonamide injection was approved by the State Food and Drug Administration for the treatment of central non-small cell lung cancer with severe airway obstruction. This is the first approved chemical ablative drug for intratumor local injection via fibrobronchoscope in China. It is also the first drug indicated to reduce severe airway obstruction in adult patients with central non-small cell lung cancer (NSCLC), filling the gap in respiratory interventional drug therapy.
The drug was developed by Academician Zhong Nanshan and submitted clinical application on November 21, 2003. The original clinical application applicant was Beijing Jiandakang New Drug Development Co., LTD. After a series of transfer of intellectual property rights and ownership, Tianjin Hongriya Pharmaceutical finally carried out the listing declaration and production. Its listing application was accepted by the CDE in 2018, and it was finally approved for listing after nearly five years of review.
