In September 2021 (as of September 30), the US FDA approved a total of 4 innovative drugs, including 3 new molecular entities and 1 biological drug. The four drugs, Livmarli, Ulipta, Tivdak, and Exkivity, have all been approved for marketing for the first time in the world.

2 new improved drugs, 35 generic drugs, of which 2 generic drugs are tentatively approved. Chinese pharmaceutical companies have obtained 4 ANDA approvals, involving Fosun Pharma's GLAND in India, Shanghai Shangyao Zhongxi Pharmaceutical, Hainan Shuangcheng Pharmaceutical, and Taiwan Ancheng Pharmaceutical. Among them, eslicarbazepine acetate of Shanghai Shangyao Zhongxi Pharmaceutical was temporarily approved.

1. Maralixibat-the first innovative treatment for childhood liver disease

On September 29, the US FDA approved Mirum Pharma Inc's innovative drug Livmarli (Maralixibat) for the treatment of cholestatic pruritus in children with Alagille syndrome (ALGS) over 1 year old. Maralixibat is the first drug approved by the FDA to treat this type of liver disease.

The European Medicines Agency is currently reviewing the marketing application of Maralixibat for the treatment of progressive familial intrahepatic cholestasis (PFIC2).

Maralixibat is an oral selective apical sodium-dependent bile acid transporter (ASBT) inhibitor. ASBT mediates the absorption of bile acids in the small intestine and helps them circulate back to the liver. Maralixibat can excrete more bile acids in the feces, prevent the accumulation of excessive bile acids, and control the extreme itching associated with cholestatic liver disease.

Alagille syndrome is a rare genetic disease. The patient’s bile ducts are congenital dysplasia, causing bile to accumulate in the liver and prevent the liver from working properly. ALGS can affect body organs such as the liver, heart, kidneys, and central nervous system. 15%-47% of patients will eventually need liver transplantation. Severe itching is the main feature of the disease and is most common in patients around 3 years old.

2. Atogepant-the first oral gepant to prevent paroxysmal migraine

On September 28, the US FDA approved AbbVie's new drug Ulipta (Atogepant) for the prevention of adult migraine. It is the first oral gepant specially developed for the preventive treatment of episodic migraine (EM).

Atogepant is an oral calcitonin gene-related peptide (CGRP) receptor antagonist (gepant), specially developed for the preventive treatment of migraine. CGRP and its receptors are expressed in areas of the nervous system related to the pathophysiology of migraine. Studies have shown that CGRP levels increase during migraine attacks, and selective CGRP receptor antagonists have clinical effects on migraine.

In the field of migraine, AbbVie is selling BOTOX (botulinum toxin A, onabotulinumtoxinA) and Ubrelvy (ubrogepant). Among them, BOTOX is the first preventive drug for adult chronic migraine approved by the US FDA, and Ubrelvy is the first oral CGRP receptor antagonist (gepant) approved by the US FDA for adult migraine (with or without Aura) acute treatment.

3. Tivdak-the first ADC therapy for cervical cancer

On September 20, the United States (FDA) accelerated the approval of Seagen's ADC drug Tivdak (Tisotumab vedotin) for the treatment of recurrent or metastatic cervical cancer. It is worth mentioning that Tivdak is the first and currently only approved antibody-drug conjugate (ADC) for cervical cancer, which is an important milestone for women with cervical cancer.

Tisotumab vedotin is an ADC drug under development that targets tissue factor (TF). The drug is designed to target the TF antigen on cancer cells and deliver the cytotoxic agent MMAE (monomethyl auristatin E) directly to the cancer in the cell. In cancer biology, TF is a protein involved in tumor signal transduction and angiogenesis. It is excessive in most cervical cancer patients and many other solid tumors (including ovarian, lung, pancreas, colorectal, and head and neck cancers). Express. Based on the high expression and rapid internalization of TF factor in many solid tumors, TF has become an ideal target for the development of ADC drugs.

4. Mobocertinib-the first oral therapy for lung cancer with EGFR exon 20 insertion mutation

On September 15, the US FDA approved Takeda’s new drug Exkivity (Mobocertinib Succinate) for the treatment of disease progression during or after previous platinum-containing chemotherapy. The FDA-approved detection method was confirmed to carry Epidermal Growth Factor Receptor (EGFR) No. 20 Adult patients with locally advanced or metastatic non-small cell lung cancer (mNSCLC) with EGFRex20ins. The FDA's approval has given this type of lung cancer patients a brand-new targeted treatment option.

Exkivity is the first and only oral therapy approved specifically to selectively target EGFRex20ins mutations. Exkivity's active pharmaceutical ingredient is mobocertinib, which is a new and potent small molecule tyrosine kinase inhibitor (TKI) designed to target EGFR and human epidermal growth factor receptor 2 (HER2) outside No. 20 with high selectivity Insertion mutations. In China and the United States, the drug has been granted Breakthrough Therapy Designation (BTD).

At present, Takeda has submitted a listing application for mobocertinib to China in July this year and was included in the priority review, which will soon bring good news to Chinese patients with this type of lung cancer.