FDA OKs New Molecular Entities from Novartis, Lilly, and Others

By Miranda Greenberg -

May 21, 2020

A roundup of approvals of new molecular entities (NMEs) from the US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER), featuring NME approvals from Novartis, Eli Lilly and Company, Seattle Genetics, Incyte, Immunomedics, Neurocrine Biosciences, and Deciphera.

Novartis Receives FDA Approval for Lung Cancer Drug Tabrecta
The FDA has approved Novartis’ Tabrecta (capmatinib) for the treatment of adult patients with non-small cell lung cancer (NSCLC) that has spread to other parts of the body.

Tabrecta is a kinase inhibitor for treating adult patients with metastatic non-small cell lung cancer whose tumors have a mutation that leads to mesenchymal-epithelial transition (MET) exon 14 skipping as detected by an FDA-approved test.

The FDA also approved Foundation Medicine’s FoundationOne CDx assay (F1CDx) as the companion diagnostic for Tabrecta to aid in detecting mutations that lead to MET exon 14 skipping in tumor tissue. F1CDx is a sequencing-based in vitro diagnostic device that is capable of detecting several mutations, including mutations that lead to MET exon 14 skipping.

Tabrecta was licensed to Novartis by Incyte in 2009. Under the agreement, Incyte granted Novartis worldwide exclusive development and commercialization rights to capmatinib and certain back-up compounds.

Source: US Food and Drug Administration and Novartis


Lilly Receives FDA Approval for Lung, Thyroid Cancer Drug Retevmo
The FDA has approved Eli Lilly and Company’s Retevmo (selpercatinib) to treat three types of tumors, non-small cell lung cancer, medullary thyroid cancer, and other types of thyroid cancers, in patients whose tumors have an alteration (mutation or fusion) in a specific gene (RET or “rearranged during transfection”). Retevmo is the first therapy approved specifically for cancer patients with the RET gene alterations, according to the FDA.

Specifically, Retevmo is approved to treat include non-small cell lung cancer (NSCLC) that has spread in adults, advanced medullary thyroid cancer (MTC) or MTC that has spread, in patients 12 and older who require systemic therapy (a treatment option that spreads across the entire body, is not targeted), and advanced RET fusion-positive thyroid cancer in those age 12 and older that requires systemic therapy that has stopped responding to radioactive iodine therapy or is not appropriate for radioactive iodine therapy. Retevmo is a selective RET kinase inhibitor and oral prescription product.

The FDA granted approval of Retevmo to Loxo Oncology, a subsidiary of Eli Lilly and Company. Lilly acquired Loxo Oncology in February 2019.

Source: US Food and Drug Administration and Eli Lilly and Company


Seattle Genetics Receives FDA Approval for Breast Cancer Drug Tukysa
The FDA has approved Seattle Genetics’ Tukysa (tucatinib) for treating HER2-positive metastatic breast cancer.

Tukysa is a kinase inhibitor to be used in combination with chemotherapy (trastuzumab and capecitabine) for the treatment of adult patients with advanced forms of HER2-positive breast cancer that can’t be removed with surgery, or has spread to other parts of the body, including the brain, and who have received one or more prior treatments.

The FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada, Health Sciences Authority (HSA, Singapore) and Swissmedic (SMC, Switzerland) on this review. This is the first Project Orbis partnership between the FDA, HSA and Swissmedic. Project Orbis is an initiative of the FDA Oncology Center of Excellence that provides a framework for concurrent submission and review of oncology products among international partners. While the FDA approved Tukysa, the application is still under review at the other agencies.

Source: US Food and Drug Administration and Seattle Genetics


Incyte Receives FDA Approval for Bile Duct Cancer Drug Pemazyre
The FDA has approved Incyte’s Pemazyre (pemigatinib) for treating certain types of previously treated, advanced cholangiocarcinoma, a rare form cancer of the bile ducts. 

Specifically, the drug was approved for patients with cholangiocarcinoma that is locally advanced (when cancer has grown outside the organ it started in, but has not yet spread to distant parts of the body) or metastatic (when cancer cells spread to other parts of the body) and who have tumors that have a fusion or other rearrangement of a gene called fibroblast growth factor receptor 2 (FGFR2). Pemazyre is a tablet that works by blocking FGFR2 in tumor cells to prevent them from growing and spreading.

The companion diagnostic for Pemazyre, Foundation Medicines’ FoundationOne CDx assay (F1CDx) is under FDA review. FoundationOne CDx is Foundation Medicine’s genomic profiling assay and broad companion diagnostic platform approved for all solid tumors. The companion diagnostic would help identify patients with FGFR2 fusions or rearrangements who may be eligible for Pemazyre.

Pemazyre is marketed by Incyte in the US. Incyte has granted Innovent Biologics, a Suzhou, China-based biopharmaceutical company, rights in China to develop and commercialize pemigatinib in hematology and oncology in Mainland China, Hong Kong, Macau and Taiwan. Incyte has retained all other rights to develop and commercialize pemigatinib outside of the US.

Incyte’s marketing authorization application seeking the approval of pemigatinib for patients with cholangiocarcinoma in the European Union has been validated by the European Medicines Agency.

Source: US Food and Drug Administration and Incyte


FDA OKs Immunomedics’ Antibody Drug Conjugate for Breat Cancer
The FDA has approved Immunomedics’ Trodelvy (sacituzumab govitecan-hziy) as a treatment for adult patients with metastatic triple-negative breast cancer who received at least two prior therapies for metastatic disease. 

Trodelvy is the lead product and the most advanced program in Immunomedics’ antibody-drug conjugate (ADC) platform. Trodelvy is an ADC that is directed against Trop-2, a cell-surface protein expressed in many solid cancers. Trodelvy binds to Trop-2 and delivers the anti-cancer drug, SN-38, to kill cancer cells. Trodelvy is currently being evaluated as a treatment for eight hard-to-treat solid cancers.

Source: US Food and Drug Administration and Immunomedics


FDA OKs Neurocrine's Parkinson’s Disease Drug Ongentys
The FDA has approved Neurocrine Biosciences’ Ongentys (opicapone) as an add-on treatment to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes.

Ongentys is an oral, selective catechol-O-methyltransferase (COMT) inhibitor that helps block the COMT enzyme that breaks down levodopa, a therapy for controlling motor symptoms in patients with Parkinson's disease. Ongentys protects levodopa by reducing its breakdown in the blood, thereby making more levodopa available to reach the brain, prolonging its clinical effects and helping patients achieve motor symptom control.

In June 2016, BIAL–Portela & CA, S.A. (BIAL), a pharmaceutical company headquartered in São Mamede do Coronado, Trofa, Portugal, received approval from the European Commission for Ongentys as an adjunct therapy to preparations of levodopa/DOPA decarboxylase inhibitors in adult patients with Parkinson's disease and end-of-dose motor fluctuations who cannot be stabilized on those combinations. BIAL currently markets Ongentys in Germany, the UK, Spain, Portugal, and Italy. Neurocrine Biosciences in-licensed opicapone from BIAL in 2017 and has exclusive development and commercialization rights in the US and Canada.  

Source: Neurocrine Biosciences


FDA OKs Deciphera's Gastrointestinal Cancer Drug Qinlock
The FDA has approved Deciphera Pharmaceuticals’ Qinlock (ripretinib) tablets as a fourth-line treatment for advanced gastrointestinal stromal tumor (GIST), a type of tumor that originates in the gastrointestinal tract. Qinlock is indicated for adult patients who have received prior treatment with three or more kinase inhibitor therapies, including imatinib.

Deciphera says it plans to make Qinlock commercially available in the US next week (as reported on May 15, 2020).

The FDA collaborated with the Australian Therapeutic Goods Administration (TGA) and Health Canada on the review of this application as part of Project Orbis, an initiative of the FDA Oncology Center of Excellence that provides a framework for concurrent submission and review of oncology products among international partners.

The review of the applications is ongoing for the Australian TGA and Health Canada.

Source: US Food and Drug Administration and Deciphera Pharmaceuticals