FDA Approval of Second Drug Based on Biomarker Leads New Molecular Entity Drug Approvals

The FDA has approved a second cancer treatment based on a biomarker, rather than tumor location. Plus, a roundup of approvals of new molecular entities by the FDA from November 20 to November 28, 2018 featuring Teva Pharmaceutical Industries, Celltrion, Pfizer, Astellas Pharma, Sobi, Novimmune, and Catalyst Pharmaceuticals.

FDA Oks Second Cancer Drug Based on Biomarker Rather Than Tumor Location
The US Food and Drug Administration (FDA) has granted Bayer and Loxo Oncology, a Stamford, Connecticut-based biopharmaceutical company, accelerated approval for Vitrakvi (larotrectinib), a treatment for adult and pediatric patients whose cancers have a specific genetic feature (biomarker). This is the second time the agency has approved a cancer treatment based on a common biomarker across different types of tumors rather than the location in the body where the tumor originated. The first treatment approved in this way was Merck & Co.’s Keytruda (pembrolizumab), the company’s anti-PD-1 therapy, an immuno-oncology drug.  

The Vitrakvi approval marks a new paradigm in the development of cancer drugs that are “tissue agnostic.” It follows the policies that the FDA developed in a guidance document released in October 2018, Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease, which provided guidance on drug development based on different molecular alterations, some of which may occur at low frequencies, that impact common proteins or pathways involved in the pathogenesis of diseases and be relevant across multiple disease types.

“Today’s [November 26,2018] approval marks another step in an important shift toward treating cancers based on their tumor genetics rather than their site of origin in the body,” said FDA Commissioner Scott Gottlieb in a November 26, 2018 agency statement. “This new site-agnostic oncology therapy isn’t specific to a cancer arising in a particular body organ, such as breast or colon cancer. Its approval reflects advances in the use of biomarkers to guide drug development and the more targeted delivery of medicine. We now have the ability to make sure that the right patients get the right treatment at the right time.”

Vitrakvi is indicated for treating adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity and have no satisfactory alternative treatments or that have progressed following treatment. Prior to the approval of Vitrakvi, there had been no treatment for cancers that frequently express this mutation.

Vitrakvi also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

In May 2017, the FDA approved a biomarker for Merck & Co.’s Keytruda (pembrolizumab). The approval was for treating adult and pediatric patients with unresectable or metastatic solid tumors that have been identified as having a biomarker referred to as microsatellite instability-high (MSI-H) or mismatch repair deficient. Tumors with these biomarkers are most commonly found in colorectal, endometrial, and gastrointestinal cancers, but also less commonly appear in cancers arising in the breast, prostate, bladder, thyroid gland and other places.

Source: FDA 


FDA OKs Pfizer’s New Leukemia Drug
The US Food and Drug Administration (FDA) has approved Pfizer’s Daurismo (glasdegib), a once-daily oral medicine, for treating newly-diagnosed acute myeloid leukemia (AML) in adult patients who are 75 years or older or who have comorbidities that preclude use of intensive induction chemotherapy.

Daurismo is a once-daily oral Hedgehog pathway inhibitor. The Hedgehog signaling pathway plays a role in embryogenesis, the process by which human embryos are developed. In adults, however, abnormal activation of this pathway is thought to contribute to the development and persistence of cancer stem cells, according to information from Pfizer.

Source: Pfizer


FDA Approves Astellas Pharma’s Leukemia Drug
The US Food and Drug Administration (FDA) has approved Astellas Pharma’s Xospata (gilteritinib) for treating adult patients who have relapsed or refractory (resistant to treatment) acute myeloid leukemia (AML) with a FLT3 mutation.

Xospata was granted both orphan-drug designation and fast-track designation by the FDA. Gilteritinib also received orphan-drug designation from the European Commission and orphan-drug designation from the Japan Ministry of Health, Labor and Welfare.

Source: Astellas Pharma


FDA OKs Sobi, Novimmune’s Rare-Disease Drug Gamifant
The US Food and Drug Administration (FDA) has approved Gamifant (emapalumab-lzsg), an antibody for treating pediatric and adult patients with primary hemophagocytic lymphohistiocytosis (HLH), an autoimmune disease, from Swedish Orphan Biovitrum (Sobi), a specialty healthcare company headquartered in Stockholm, Sweden and Novimmune, a Geneva, Switzerland-based biopharmaceutical company focused on antibody-based medicines.

Primary HLH is an ultra-rare syndrome of hyperinflammation with high morbidity and mortality and for which there was previously no approved drug.

In the US, Gamifant was reviewed under priority review and received orphan-drug designation, breakthrough-therapy designation, and rare pediatric disease designation from the FDA. Novimmune is eligible to receive a priority review voucher (PRV) with the approval. In Europe, emapalumab was accepted for review by the European Medicines Agency (EMA) in August 2018 and has been granted orphan-drug designation and PRIority MEdicine (PRIME) status by the EMA.

Gamifant was developed and submitted for approval to the FDA by Novimmune. Sobi acquired the global rights to Gamifant from Novimmune through an exclusive licensing agreement announced in July 2018.

Source: Novimmune


FDA OKs Catalyst Pharma’s Rare-Disease Drug
The US Food and Drug Administration (FDA) has approved Firdapse (amifampridine), a drug for treating adults with Lambert-Eaton Myasthenic Syndrome (LEMS), an autoimmune disease, from Catalyst Pharmaceuticals, a Coral Gables, Florida-headquartered biopharmaceutical company focused on rare diseases.

LEMS is a rare autoimmune disease that affects approximately 1 in 100,000 people in the US, according to information from Catalyst Pharma. LEMS is a rare autoimmune disorder, most often characterized by fatigable limb muscle weakness. The disease is caused by autoantibodies against voltage-gated calcium channels located in the nerve-muscle junction, resulting in improper nerve-muscle communication, leading to progressive muscle weakness when left untreated, according to information from Catalyst Pharma.

Firdapse 10-mg tablets is an oral, nonspecific, voltage-dependent, potassium (K+) channel blocker that causes depolarization of the presynaptic membrane and slows or inhibits repolarization. Firdapse is approved in the US and the European Union for use by patients with LEMS. Firdapse is expected to be commercially available early in the first quarter of 2019.

Source: Catalyst Pharma

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