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A collection of recent regulatory approvals and other actions globally

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In news that marks a significant breakthrough moment for cannabis-based therapeutics, London-based GW Pharmaceuticals plc and its U.S. subsidiary Greenwich Biosciences announced this summer that the U.S. Food and Drug Administration (FDA) had approved Epidiolex (cannabidiol) oral solution for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome in patients two years of age or older.
Epidiolex is the first prescription pharmaceutical formulation of highly purified, plant-derived cannabidiol (CBD), a cannabinoid lacking the high associated with marijuana, and the first in a new category of anti-epileptic drugs. Product availability is pending rescheduling, which was expected to occur within 90 days of the approval.
“Today’s approval of Epidiolex is a historic milestone, offering patients and their families the first and only FDA-approved CBD medicine to treat two severe, childhood-onset epilepsies,” said Justin Gover, GW’s CEO.
LGS and Dravet syndrome, which develop in childhood, are rare, severe forms of epilepsy that are notoriously treatment-resistant.
“For those living with intractable seizures caused by LGS and Dravet syndrome, Epidiolex represents a true medical advancement,” said Philip Gattone, president and CEO of the Epilepsy Foundation. “Clinical development for these rare and severe conditions is essential, and today’s news brings hope for these patients and their families that a new treatment option may have the potential to help better control their seizures.”
Epidiolex will be marketed in the United States by Greenwich Biosciences. Outside the United States, this medicine is currently under review by the European Medicines Agency for the treatment of seizures associated with LGS and Dravet Syndrome, and the European decision on whether to recommend approval is expected in the first quarter of 2019.

Europe OKs expanded use for Sprycel
Jumping over to Europe, the European Commission recently approved the use of Bristol-Myers Squibb’s Sprycel (dasatinib) to treat children and adolescents aged 1 to 18 years with Philadelphia chromosome-positive chronic myeloid leukemia (CML) in chronic phase, including a powder formulation for oral suspension.
“Treatment options for pediatric patients with CML are limited, as are formulations that correspond with the unique demands of children with cancer. Our decision to pursue an expanded indication for Sprycel in this new patient population and as a new formulation is indicative of our commitment to extending the potential of our medicines to address the unmet needs of patients with cancer, regardless of the incidence of the disease,” said Bristol-Myers Squibb Head of Oncology Development Dr. Fouad Namouni.

Two CAR-T cell therapies poised for approval in Europe
Sticking with Europe for a moment, but not quite yet in approval mode, the European Medicines Agency (EMA) recently recommended the first two marketing authorizations for CAR-T cell medicines in the European Union. Those medicines, Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel), are advanced therapies for blood cancer.
Kymriah and Yescarta are also the first drugs supported through EMA’s PRIority MEdicines (PRIME) scheme to receive positive opinions from the Committee for Medicinal Products for Human Use. The voluntary scheme provides early and enhanced scientific and regulatory support to medicines that have the potential to address, to a significant extent, patients’ unmet medical needs. Kymriah was granted eligibility to PRIME on June 23, 2016, for the treatment of acute lymphoblastic leukaemia, while Yescarta was granted eligibility to PRIME on May 26 of that same year for the treatment of diffuse large B-cell lymphoma.
“CAR-T cells transform the fight against serious and often fatal diseases in the EU,” said Dr. Martina Schüssler-Lenz, chair of the Committee for Advanced Therapies. “Kymriah and Yescarta offer an innovative approach where patients’ cells are reprogrammed and reinjected to attack the cancer.”

A first against smallpox
Back in the United States, SIGA Technologies Inc., a commercial-stage pharmaceutical company focused on the health security market, recently announced the FDA approval of oral TPOXX (tecovirimat) for the treatment of smallpox to mitigate the impact of a potential outbreak. TPOXX is a small-molecule antiviral treatment for smallpox and marks the first therapy specifically approved for this indication.
FDA approval was based on data from 12 clinical trials of oral TPOXX in over 700 healthy human volunteers, which showed no drug-related serious adverse events. Four pivotal trials in non-human primates and two pivotal trials in rabbits demonstrated that TPOXX significantly reduced both mortality and viral load in non-human primates infected with monkeypox virus and in rabbits infected with rabbitpox virus.
“The FDA approval of TPOXX achieves an important objective for both SIGA and our lead partner in the U.S. government, the Biomedical Advanced Research and Development Authority,” said Dr. Phil Gomez, CEO of SIGA. “The approval validates this novel smallpox therapy as an important medical countermeasure in response to a potential smallpox outbreak.”
Smallpox remains a significant bioterrorism threat, and the U.S. government has been focused on the development of medical countermeasures to mitigate that threat. Vaccines are an important component of a response to a smallpox outbreak, but the availability of a therapeutic such as TPOXX is critical to reduce the morbidity and mortality of such an outbreak.

FDA allows Azedra to move forward
On July 30, Progenics Pharmaceuticals Inc. announced that the FDA had approved the New Drug Application for Azedra (iobenguane I 131) 555 MBq/mL injection for intravenous use. Azedra, a radiotherapeutic, is indicated for the treatment of adult and pediatric patients 12 years and older with iobenguane scan-positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy. Azedra is the first and only approved therapy for this indication.
Pheochromocytoma and paraganglioma are neuroendocrine cancers that arise from cells in and around the adrenal glands. Pheochromocytoma and paraganglioma tumors frequently secrete high levels of hormones that can lead to life-threatening high blood pressure, heart failure and stroke in these patients.
“Azedra is a true breakthrough in treating pheochromocytoma and paraganglioma, delivering an effective anticancer therapy to these tumors,” remarked Dr. Daniel Pryma, chief of the Division of Nuclear Medicine & Clinical Molecular Imaging at the Perelman School of Medicine at the University of Pennsylvania, the trial’s lead investigator. “With this innovative, rationally designed treatment, we finally have a therapeutic option that helps address patients’ needs.”
“As the first FDA approved therapy for unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy, Azedra provides a new treatment option for physicians and their patients,” said Mark Baker, CEO of Progenics. “AZEDRA has been shown to decrease the need for blood pressure medication and reduce tumor size in some patients.”

An NDA approval for efti
Australia-based Immutep Ltd. recently announced the approval of its Investigational New Drug application by the FDA for eftilagimod alpha (efti or IMP321), a LAG-3Ig fusion protein. This allows the company, subject to the completion of other preparatory steps, to initiate the TACTI-002 Phase 2 clinical study in the United States that will evaluate the combination of efti and anti-PD-1 therapy Keytruda (pembrolizumab) in patients with non-small cell lung carcinoma or head and neck carcinoma. Immutep expects to commence the TACTI-002 trial in the second half of 2018 and to report the first data from the trial in 2019.
“This is one more important milestone for the company’s pipeline of LAG-3 immunotherapeutic products that aim to transform the treatment of cancer and autoimmune diseases. Through our clinical programs, and the efforts of our partners, we believe Immutep is securely positioned to play an important role in the development of combination therapies utilizing LAG-3,” commented Marc Voigt, CEO of Immutep.

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