For your approval

A collection of recent regulatory approvals and other actions globally

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As this issue was coming together, the scuttlebutt was that Pfizer and Allergan were going to have to scuttle their planned $160-billion merger, so why don’t we start off with some good news for Pfizer on the regulatory front before we progress with the rest of the roundup.
On March 14, Pfizer Inc. announced that the U.S. Food and Drug Administration (FDA) had approved a supplemental New Drug Application for Xalkori (crizotinib) to treat patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are ROS1-positive.
In 2015, the FDA granted Breakthrough Therapy and Priority Review designations for this indication. Xalkori had already been approved for the indication of patients with metastatic NSCLC whose tumors are anaplastic lymphoma kinase (ALK)-positive as detected by an FDA-approved test.
“[This] approval of Xalkori for ROS1-positive metastatic NSCLC represents an important milestone for patients who previously had limited treatment options,” said Dr. Mace Rothenberg, senior vice president of clinical development and medical affairs and chief medical officer for Pfizer Oncology. “As the only FDA-approved biomarker-driven therapy that includes two distinct molecular targets in metastatic NSCLC, ROS1 and ALK, Xalkori exemplifies our commitment to precision drug development and to identifying the right treatment for the right patient.”
An FDA-approved test for the detection of ROS1 rearrangements in NSCLC is not currently available; however, laboratory developed tests are available. A companion diagnostic test is currently under development to identify patients with ROS1-positive metastatic NSCLC who may benefit from treatment with Xalkori.
Additionally, the European Medicines Agency (EMA) is reviewing an application to extend the marketing authorization of Xalkori to include the treatment of adult patients with ROS1-positive advanced NSCLC. In Europe, Xalkori is indicated for the first-line treatment of adults with ALK-positive advanced NSCLC and for the treatment of adults with previously treated ALK-positive advanced NSCLC.

Inflectra becomes first biosimilar mAb licensed in U.S.
INCHEON, Republic of Korea—Early April saw Celltrion, a global biopharmaceutical company, announce that the FDA had approved Inflectra (biosimilar infliximab) for all indications of the reference product, Janssen Biotech Inc.'s Remicade (infliximab). Inflectra is the first biosimilar monoclonal antibody (mAb) medication to receive approval in the United States, and is indicated for the treatment of patients with a number of serious autoimmune diseases, including Crohn's disease, ulcerative colitis, rheumatoid arthritis, ankylosing spondylitis, psoriatic arthritis and plaque psoriasis.
“The FDA approval of Inflectra, just the second biosimilar to be approved in the U.S., is significant for the medical community as this therapy has conclusively demonstrated comparable safety and efficacy to the reference product, and will provide the medical community with an alternative, more affordable treatment option,” said Dr. Vibeke Strand, adjunct clinical professor in the Division of Immunology/Rheumatology at the Stanford University School of Medicine. “This approval will help to remove barriers for the many healthcare professionals and their patients where cost and access to treatment for these chronic autoimmune diseases has been a challenge.”
“As one of the first companies to navigate the biosimilar approval pathway with the FDA, we believe this approval will be an essential step in helping to clarify the application process for these critical medicines,” said HyoungKi Kim, CEO of Celltrion. “Our experience with biosimilars outside the U.S. suggests that Inflectra provides patients with both therapeutic and financial benefits, and we hope to see the same value provided in the U.S.”

New treatment for patients with multiple myeloma
HORSHAM, Pa.—Janssen Biotech recently got a nod from the EMA that recommended granting a conditional marketing authorization for Darzalex (daratumumab) for the treatment of adults with relapsed and refractory multiple myeloma. Darzalex is to be used in patients whose previous treatment included a proteasome inhibitor and an immunomodulatory agent (other types of cancer medicines) and whose disease worsened after treatment.
Darzalex is a monoclonal antibody that works by activating the body’s immune system to attack and kill multiple myeloma cells.
The Committee for Medicinal Products for Human Use (CHMP) reviewed Darzalex under EMA’s accelerated assessment program and recommended conditional approval for the medicine. As part of the conditional marketing authorization, Janssen Biotech must provide results from two Phase 3 studies of Darzalex used in combination with standard treatments for this disease (lenalidomide/dexamethasone and bortezomib/dexamethasone). Both studies are ongoing and the data will be provided by the applicant by the second half of 2017.

FDA approves new formulation of Makena
WALTHAM, Mass.—AMAG Pharmaceuticals Inc. recently announced that the FDA had approved the company’s single-dose, preservative-free formulation of Makena (hydroxyprogesterone caproate injection). Makena is the only FDA-approved treatment indicated to reduce the risk of preterm birth in women who are pregnant with one baby and who have spontaneously delivered one preterm baby in the past.
The single-dose formulation is an expansion of the Makena product line. Packaged in trays of four (a month’s supply), the single-dose formulation provides an alternative package size to the current multidose vial, which contains five weekly injections. AMAG currently expects to commercialize the new single-dose, preservative-free formulation of Makena in the second quarter of 2016.

First oral treatment for Fabry disease recommended for approval in EU
CRANBURY, N.J.—Amicus Therapeutics shared news in early April that the EMA had recommended granting a marketing authorization in the EU for Galafold (migalastat) for the treatment of Fabry disease, a rare genetic disorder.
Patients with Fabry disease do not have enough of an enzyme called alpha-galactosidase A. This enzyme normally breaks down a fatty substance called globotriaosylceramide (GL-3). If the enzyme is missing or is defective, GL-3 is not broken down and it builds up in the body’s cells, such as heart and kidney cells. Patients with Fabry disease may have a wide range of signs and symptoms, including severe conditions such as kidney failure, heart problems and increased risk of strokes.
Currently, the standard treatment for this disease is enzyme replacement therapy (ERT), which consists of an intravenous infusion (drip) of a copy of the missing enzyme. Galafold is the first oral treatment for Fabry disease and may provide a more convenient treatment option for patients.

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