June FDA Decision Watch: Keytruda, Revlimid Among 11 Drugs Facing Regulatory Verdicts

Renowned financial news website RTTNews recently published an article stating that 11 drugs will receive important regulatory review decisions in the United States in June. Below is an introduction to each drug:

01 Zerbaxa

On June 3, the FDA will issue a review decision on Merck’s supplemental new drug application (sNDA) for Zerbaxa, a combination antibiotic, for the treatment of hospital-acquired pneumonia (HAP) in adult patients, including hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia caused by susceptible Gram-negative bacteria.

This drug is a combination antibiotic product administered via intravenous infusion, composed of the novel cephalosporin antibiotic ceftolozane and the β-lactamase inhibitor tazobactam.

Zerbaxa was acquired by MSD following its $9.5 billion acquisition of Cubist in 2014. In the U.S. market, Zerbaxa was approved in late 2014, and its current indications are:

• For the treatment of complicated urinary tract infections (including pyelonephritis) caused by susceptible Gram-negative bacteria in adult patients;

• In combination with metronidazole, for the treatment of complicated intra-abdominal infections in adult patients caused by susceptible Gram-negative and Gram-positive bacteria.

On June 6, the FDA Advisory Committee will review pretomanid tablets. The drug is indicated for use in combination with bedaquiline and linezolid to treat adults with pulmonary extensively drug-resistant tuberculosis (XDR-TB), or those who are intolerant to or nonresponsive to multidrug-resistant tuberculosis (MDR-TB) treatment. The FDA will make a final review decision in the third quarter of 2019.

Tuberculosis is an airborne disease transmitted through coughing or sneezing and is a leading cause of infectious diseases worldwide. Caused by Mycobacterium tuberculosis, it is difficult to treat. According to data from the Stop TB Partnership, there were 558,000 cases of drug-resistant tuberculosis in 2018.

03 Keytruda

On June 10, the FDA will issue a review decision on the supplemental Biologics License Application (sBLA) for Merck’s Keytruda as a monotherapy or in combination with chemotherapy for the first-line treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck. Additionally, on June 17, the FDA will issue a review decision on the sBLA for Keytruda as a monotherapy for patients with advanced small cell lung cancer who have experienced disease progression after receiving at least two prior therapies.

In 2018, global sales of Keytruda reached $7.17 billion, and it is projected to surpass Opdivo starting this year to become the world’s best-selling PD-(L)1 cancer immunotherapy.

04 Gvoke HypoPen

On June 10, the FDA will issue a review decision on Xeris Pharmaceuticals’ New Drug Application (NDA) for Gvoke HypoPen for the treatment of severe hypoglycemia. The drug delivers ready-to-use glucagon via a commercial auto-injector. If approved, Gvoke HypoPen will become the first and only ready-to-use, room-temperature-stable liquid formulation of glucagon for the treatment of severe hypoglycemia.

Xeris believes that approximately 3.5 million people are clinically eligible for glucagon use. Following the launch of Gvoke HypoPen in the United States, its annual peak sales are projected to reach $2 billion.

05 Vyleesi

On June 23, the FDA will make a review decision on AMAG Pharmaceuticals’ Vyleesi for the treatment of hypoactive sexual desire disorder (HSDD) in premenopausal women.

HSDD is the most common type of female sexual dysfunction, characterized by low sexual desire that causes marked distress or interpersonal difficulty. In the United States, HSDD affects approximately 15 million women, of whom about 5.8 million are premenopausal.

Currently, only one drug has been approved for the treatment of hypoactive sexual desire disorder (HSDD) in premenopausal women: Addyi, developed by Sprout Pharmaceuticals and also known as the “female Viagra.” In January 2017, Palatin Technologies obtained exclusive North American rights to develop and commercialize Vyleesi. Fosun Pharma holds the exclusive commercialization rights for Vyleesi in the Chinese market.

AMAG estimates that Vyleesi could generate $1 billion in sales after its market launch.

06 Edsivo

On June 25, the FDA will issue a review decision on Acer Pharmaceuticals’ New Drug Application (NDA) for Edsivo, a drug indicated for the treatment of patients with vascular Ehlers-Danlos syndrome (vEDS) confirmed by genetic testing to carry mutations in the type III collagen gene (COL3A1). The FDA had previously granted orphan drug designation to Edsivo for the treatment of vEDS.

Edsivo is a beta-blocker, a new chemical entity initially developed for the treatment of hypertension. It is believed to provide clinical benefits to patients with vascular Ehlers-Danlos syndrome (vEDS) by promoting normal collagen synthesis in blood vessels and by redirecting mechanical stress away from vessels most prone to dissection and rupture. Although indicated for the treatment of mild-to-moderate hypertension, celiprolol does not lower blood pressure in individuals with normal blood pressure. If approved for marketing, Edsivo is projected to achieve sales of $400 million.

07 Dupixent

On June 26, the FDA will issue a review decision on Regeneron’s supplemental Biologics License Application (sBLA) for Dupixent, a novel anti-inflammatory drug, as an add-on maintenance treatment for adult patients with severe chronic rhinosinusitis with nasal polyps whose disease is not adequately controlled. In March this year, this indication received a positive opinion from the EU Committee for Medicinal Products for Human Use (CHMP), recommending approval.

Dupixent, co-developed by Regeneron and Sanofi, was approved in March 2017 for moderate-to-severe atopic dermatitis and in October 2018 for moderate-to-severe asthma. Its sales in 2018 amounted to $922 million.

08 Revlimid

On June 27, the FDA will issue a review decision on Celgene’s sNDA for Revlimid in combination with rituximab for the treatment of patients with previously treated follicular and marginal zone lymphoma.

Revlimid is an immunomodulatory agent whose active pharmaceutical ingredient is lenalidomide, a next-generation derivative of thalidomide. It lacks teratogenic toxicity, exhibits potency 100 times greater than that of thalidomide, and possesses immunomodulatory, anti-angiogenic, and antineoplastic properties. This drug is indicated for the treatment of multiple conditions, including multiple myeloma, myelodysplastic syndromes, and mantle cell lymphoma.

Revlimid is a product on which Celgene heavily relies, maintaining double-digit growth in recent years. Its global sales reached $9.68 billion in 2018 and are projected to reach $10.8 billion in 2019. Another drug from Celgene, Pomalyst/Imnovid (pomalidomide), achieved sales of $2.04 billion in 2018, representing a 26.4% increase compared to the same period last year.

09 Soliris

On June 28, the FDA will issue a review decision on Alexion’s supplemental Biologics License Application (sBLA) for Soliris, a blockbuster drug, in the treatment of neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a rare, devastating, complement-mediated autoimmune disease of the central nervous system that primarily affects the optic nerves and spinal cord, leading to blindness and paralysis. Currently, there are no approved therapies for NMOSD.

Soliris is a first-in-class complement inhibitor that works by inhibiting the C5 protein in the terminal part of the complement cascade. The complement cascade is part of the immune system, and its uncontrolled activation plays an important role in rare diseases.

Soliris was approved for marketing in 2007, and its current indications include: paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and anti-acetylcholine receptor antibody-positive myasthenia gravis. The drug is also one of the best-selling orphan drugs globally, with sales reaching $3.563 billion in 2018.

10 New Thiola Formula

On June 30, the FDA will issue a review decision on the New Drug Application (NDA) for the new formulation of Thiola for the treatment of cystinuria, a rare hereditary disorder of cystine transport that leads to elevated cystine levels in the urine and the formation of recurrent kidney stones.

In 1988, the FDA approved 100-mg tablet formulations of Thiola for the treatment of cystinuria. In May 2014, Retrophin acquired the U.S. marketing rights to Thiola from the privately held Mission Pharmacal.

11 DOPTELET

On June 30, the FDA will issue a review decision on Dova Pharmaceuticals’ supplemental New Drug Application (sNDA) for Doptelet in the treatment of patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to previous therapies.

ITP is an autoimmune bleeding disorder characterized by thrombocytopenia, such as abnormally low platelet counts. Doptelet is a second-generation oral thrombopoietin receptor agonist (TPO-RA) that mimics the action of thrombopoietin (TPO), the primary regulator of normal platelet production.

Doptelet was approved by the FDA in May 2018 for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo surgery, with sales reaching $7.7 million in 2018. (Compiled by Sina Medicine/newborn)

Reference Source: Biotech Stocks Facing FDA Decision In June 2019