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A collection of recent regulatory approvals and actions globally

Jeffrey Bouley
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From Novartis to Genentech, Bristol-Myers Squibb to True North and Merck to UCB, we give you this month a small sampling of the regulatory activities in Europe and America regarding therapeutics for a wide range of conditions.

Breakthrough status for Novartis’ PKC412
EAST HANOVER, N.J.— Novartis announced in February that the U.S. Food and Drug Administration (FDA) had granted Breakthrough Therapy designation to PKC412 (midostaurin), an investigational treatment for adults with newly diagnosed acute myeloid leukemia (AML) who are FLT3 mutation-positive, as detected by an FDA-approved test, and who are eligible to receive standard induction and consolidation chemotherapy.
The designation is primarily based upon the positive results from the Phase 3 RATIFY (CALGB 10603) clinical trial. Patients who received PKC412 and standard induction and consolidation chemotherapy experienced a significant improvement in overall survival compared to those who received standard induction and consolidation chemotherapy alone.
“For more than 25 years, medical developments have been limited for AML patients, and the chemotherapy treatment strategy has essentially remained unchanged,” said Dr. Alessandro Riva, global head of Novartis Oncology Development and Medical Affairs. “We look forward to working closely with the FDA to bring PKC412, the first potential AML targeted therapy, to patients as quickly as possible.”

FDA approval for Genentech’s Gazyva
SOUTH SAN FRANCISCO, Calif.—Genentech, a member of the Roche Group, announced late February that the FDA had approved Gazyva (obinutuzumab) plus bendamustine chemotherapy followed by Gazyva alone as a new treatment for people with follicular lymphoma who did not respond to a Rituxan (rituximab)-containing regimen, or whose follicular lymphoma returned after such treatment. Follicular lymphoma is the most common type of indolent non-Hodgkin’s lymphoma (NHL) and accounts for approximately one in five cases of NHL.
“People with follicular lymphoma whose disease returns or worsens despite treatment with a Rituxan-containing regimen need more options because the disease becomes more difficult to treat each time it comes back,” said Dr. Sandra Horning, Genentech’s chief medical officer and head of global product development. “Gazyva plus bendamustine provides a new treatment option that can be used after relapse to significantly reduce the risk of progression or death.”
The approval is based on results from the Phase 3 GADOLIN study, which showed that, in people with follicular lymphoma whose disease progressed during or within six months of prior Rituxan-based therapy, Gazyva plus bendamustine followed by Gazyva alone demonstrated a 52-percent reduction in the risk of disease worsening or death compared to bendamustine alone.

Opdivo gets new indication
NEW YORK—Bristol-Myers Squibb’s Opdivo (nivolumab) expanded its reach when the European Medicines Agency (EMA) recommended recently extending its use to include the treatment of adult patients with advanced renal cell carcinoma who have received prior therapy. Opdivo was first authorized in the European Union in June 2015 for the treatment of advanced melanoma and already had its use extended (in October 2015) to treatment of the advanced stages squamous non-small cell lung cancer.
Renal cell carcinoma is the most common form of kidney cancer in adults. Advanced renal cell carcinoma includes both metastatic disease and locally advanced renal cell carcinoma that cannot be resected. Patients with advanced renal cell carcinoma have a poor long-term prognosis; therefore, new medicines are urgently needed for patients.

Orphan status for True North’s TNT009
SOUTH SAN FRANCISCO, Calif.—True North Therapeutics, a clinical-stage biotechnology company developing novel therapies for complement-mediated rare diseases, recently announced that the EMA had granted Orphan Drug designation for TNT009 for the treatment of autoimmune hemolytic anemia, including cold agglutinin disease (CAD), in which autoantibodies target and destroy red blood cells, causing anemia, fatigue and potentially fatal thrombosis.
“Receiving Orphan Drug designation from the EMA is another important step forward for True North as we continue to advance development of TNT009,” said Dr. Nancy Stagliano, CEO of True North. “We recently initiated a Phase 1b clinical study evaluating TNT009 in patients with cold agglutinin disease and look forward to reporting top-line data later this year.”

Zepatier gets nod for chronic HCV
KENILWORTH, N.J.—Merck, known as MSD outside the United States and Canada, recently announced FDA approval of Zepatier (elbasvir and grazoprevir) for the treatment of adult patients with chronic hepatitis C virus (HCV) genotype (GT) 1 or GT4 infection, with or without ribavirin.
The FDA previously granted two Breakthrough Therapy designations to Zepatier, for the treatment of chronic HCV GT1 infection in patients with end-stage renal disease on hemodialysis and for the treatment of patients with chronic HCV GT4 infection. Across multiple clinical studies, Zepatier achieved high rates of sustained virologic response, ranging from 94 to 97 percent in GT1-infected patients and 97 to 100 percent in GT4-infected patients.

FDA gives approval to Briviact
BRUSSELS, Belgium—Feb. 19 saw UCB announce that the FDA had approved Briviact (brivaracetam) as adjunctive therapy in the treatment of partial-onset seizures in patients 16 years of age and older with epilepsy.
“The FDA’s approval of Briviact is significant, because uncontrolled seizures can have serious, long-term effects, and approximately 30 percent of epilepsy patients remain uncontrolled on currently available treatments,” said Dr. Pavel Klein, director of the Mid-Atlantic Epilepsy and Sleep Center in Bethesda, Md.
“We are excited to introduce Briviact as a new therapeutic option that may make a difference in the lives of people with epilepsy in the U.S.,” said Jeff Wren, head of neurology and executive vice president at UCB. “This approval is the culmination of more than eight years of clinical trials involving more than 2,400 adult patients with partial-onset seizures. The development of Briviact builds upon our longstanding heritage in developing meaningful treatment solutions for people living with epilepsy.”

Jeffrey Bouley

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