8 new drugs approved by the FDA in February 2021

In February 2020, the FDA approved a total of 8 new drugs (excluding vaccines), including 6 new molecular entities, 1 monoclonal antibody biological product, and 1 cell therapy.

1. Tepmetko (Tepotinib)

On February 3, the FDA approved the MET inhibitor tepotinib (trade name: TEPMETKO) developed by EMD Serono, a subsidiary of Merck, Germany through a priority review method for the treatment of jumping mutations in exon 14 (MET ex14) of the MET gene. Of adult patients with metastatic non-small cell lung cancer (NSCLC), regardless of whether these patients have been treated before. Tepotinib is the first and only once-daily oral MET inhibitor approved by the FDA.

Tepotinib is independently developed by Merck, Germany, and can inhibit the oncogenic MET receptor signal caused by mutations in the MET gene. It was approved for marketing in Japan as early as March 2020. It is the world's first oral MET inhibitor approved by regulatory authorities for the treatment of advanced NSCLC with MET gene mutations. The FDA has previously granted tepotinib breakthrough therapy designation and approved its listing through the Real-Time Oncology Review (RTOR) program. The FDA also recently granted orphan drug designation (ODD) for tepotinib.

In China, Merck submitted a clinical application to NMPA as early as July 2013, and so far has carried out 4 clinical studies in China.

2. Breyanzi (lisocabtagene maraleucel)

On February 5, the FDA approved the listing of the CAR-T therapy Breyanzi (lisocabtagene maraleucel) developed by Juno Therapeutics under Bristol-Myers Squibb (BMS) for the treatment of adult relapsed or refractory large b-cell lymphoma. Breyanzi is a CAR-T cell therapy targeting CD19 antigen and the fourth CAR-T therapy approved by the FDA for marketing.

Breyanzi is an autologous CAR-T therapy targeting CD19 antigen. It has been awarded breakthrough therapy designation and regenerative medicine advanced therapy designation (RMAT) granted by FDA. The uniqueness of this therapy is that the ratio of CD8-positive and CD4-positive T cells in CAR-T therapy is controlled, which can better control the side effects of cell therapy.

3. Ukoniq (umbralisib)

On February 5, the FDA approved the listing of TG company umbralisib (trade name UKONIQ) for the treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who had received at least one anti-CD20-based treatment before and at least received it before Adult patients with relapsed or refractory follicular lymphoma (FL) who have received third-line systemic therapy.

Umbralisib is the first and only once-daily oral phosphoinositide 3-kinase (PI3K) δ and casein kinase 1 (CK1) ε inhibitor approved to be marketed. PI3K-δ plays an important role in supporting cell proliferation and survival, cell differentiation, cell-to-cell transport and immunity, and is expressed in normal and malignant b cells. CK1-ε is an oncoprotein translation regulator, which is related to the pathogenesis of cancer cells including lymphoid malignancies. Previously, umbralisib has been approved as a breakthrough therapy by the FDA for the treatment of MZL indications, and the marketing application has also been included in the priority review. Umbralisib has also been granted Orphan Drug Designation (ODD) for the treatment of MZL and FL.

4. Evkeeza (evinacumab)

On February 11, the FDA approved the listing of regenerant Evkeeza (evinacumab) as an adjuvant therapy to other lipid-lowering drugs for the treatment of homozygous familial hypercholesterolemia (HoFH) in adults and children over 12 years of age.

The mechanism of action of Evkeeza is different from the existing HoFH therapeutic drugs. In 2017, it was granted a breakthrough drug qualification for the treatment of HoFH by the FDA. It is also the first FDA-approved targeting angiopoietin-like 3 (angiopoietin-like 3, ANGPTL3). )medicine. ANGPTL3 is a protein secreted by the liver. It is mainly expressed in the liver of humans and mice. It is named for its characteristic structure of the angiogenin family. It plays a key role in lipid metabolism and is related to lipid metabolism disorders and coronary heart disease. It is related to diseases such as atherosclerosis, diabetes, metabolic syndrome, kidney disease and liver cancer.

Evkeeza is administered by intravenous injection, once a month, and the dosage is calculated based on body weight (15mg/kg). Therefore, the annual treatment cost of each person is different according to the WAC price, which is about 450,000 US dollars per year on average. Regeneron expects to provide assistance to eligible patients through the myRARE™ project. Under this project, eligible patients covered by commercial insurance have the opportunity to receive free treatment.

5. Cosela (trilaciclib)

On February 12, the FDA approved the launch of Costela (Trilaciclib) injection developed by G1 Therapeutics to prevent bone marrow suppression caused by Platinum/etoposide regimen or topotecan regimen in adult patients with diffuse small cell lung cancer.

Trilaciclib has been granted breakthrough therapy qualification by the FDA and is the world's first and only product that is administered prophylactically before the start of chemotherapy to protect the bone marrow and immune system function.

Trilaciclib is a First-in-Class short-acting small molecule CDK 4/6 inhibitor. Prophylactic use of Trilaciclib before chemotherapy can temporarily block bone marrow cells in the G1 phase of the cell cycle and significantly reduce the killing of bone marrow cells by chemotherapy drugs. , To protect bone marrow cells and immune system function, its significance is not only to improve the quality of life of patients, but also to increase the number of chemotherapy cycles for some patients, and improve refractory tumors such as small cell lung cancer (SCLC) and triple negative breast cancer (TNBC) The overall survival benefit of the patient.

6. Amondys 45 (casimersen)

On February 25, the FDA approved Sarepta's antisense oligonucleotide drug AMONDYS 45 (casimersen) for the treatment of patients with Duchenne muscular dystrophy (DMD) with skipping mutations in exon 45. This is the third antisense oligonucleotide therapy approved by Sarepta.

AMONDYS 45 is developed by Sarepta's proprietary phosphodiamide morpholine oligomer (PMO) and exon skipping technology platform. AMONDYS 45 binds to exon 45 of the dystrophin precursor mRNA to exclude or "skip" this exon during mRNA processing. The result is a shortened, functional dystrophin protein.

7. NULIBRY (fosdenopterin)

On February 26, the FDA approved the listing of Nulibry (fosdenopterin) developed by Origin Biosciences, a subsidiary of BridgeBio, to reduce the risk of death due to type A molybdenum cofactor deficiency (MoCD). fosdenopterin is the first innovative treatment approved by the FDA to treat the disease. Molybdenum cofactor A deficiency is a rare, inherited metabolic disease that usually occurs in infants a few days after birth and can Lead to intractable seizures, brain damage, and death. A total of less than 150 patients worldwide are affected by this disease, and the median survival time of patients is 4 years.

fosdenopterin is a substrate replacement therapy that provides an exogenous source of cPMP (cyclopyran monophosphate). cPMP can be converted into molybdenum purine, and molybdenum purine can be converted into molybdenum cofactor to prevent the lack of molybdenum cofactor The resulting reduction in the synthesis of sulfite oxidase leads to the accumulation of toxic sulfites, thereby alleviating the central nervous system symptoms of infants and young children A MoCD. Nulibry has received priority review qualification, breakthrough therapy designation and orphan drug designation granted by the FDA.

8. PEPAXTO (melphalan flufenamide)

On February 26, the FDA accelerated the approval of Oncopeptides company Pepaxto (melphalan flufenamide, also known as melflufen) to be marketed in combination with Dexamethasone for the treatment of adult patients with relapsed/refractory multiple myeloma (MM). These patients have received at least four previous treatments and are resistant to at least one proteasome inhibitor, one immunomodulator and one CD38 monoclonal antibody. Pepaxto is the first peptide drug conjugate (PDC) approved by the FDA.

PEPAXT is a first in class PDC, an active, lipophilic and optimized derivative of melphalan. It can target aminopeptidase and quickly release alkylating agent into tumor cells. Melflufen is quickly absorbed by myeloma cells due to its high lipophilicity, and is immediately hydrolyzed by peptidase to selectively release the alkylating agent melphalan. Aminopeptidase is overexpressed in tumor cells, especially in advanced cancers and hypermutation tumors. In in vitro experiments, Pepaxto can increase the concentration of the alkylating agent in the cell, and its ability to kill MM cells is 50 times higher than that of the alkylating agent it carries.
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