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A collection of recent regulatory approvals and other actions globally

Jeffrey Bouley
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We begin this roundup of regulatory news with word that AbbVie’s Humira (adalimumab) received U.S. Food and Drug Administration (FDA) approval in June for the treatment of non-infectious intermediate, posterior and panuveitis, making it the first FDA-approved non-corticosteroid therapy available for adults with non-infectious intermediate, posterior and panuveitis. This approval marks the 10th approved indication for Humira in the United States for immune-mediated diseases.
The same month, the European Commission (EC) approved Humira in the European Union (EU) for the treatment of non-infectious intermediate, posterior and panuveitis in adult patients who have had an inadequate response to corticosteroids, in patients in need of corticosteroid-sparing or in whom corticosteroid treatment is inappropriate.
“We are pleased to provide patients with the first FDA-approved non-corticosteroid treatment option for certain types of uveitis, an eye disease that can flare and impact vision,” said Dr. Mike Severino, executive vice president for research and development and chief scientific officer at AbbVie. “These approvals reflect our ongoing focus on continuing to innovate with Humira to address critical unmet needs of patients living with serious immune-mediated diseases.”
Prior to this approval, ophthalmologists and rheumatologists had no FDA-approved treatment options other than corticosteroids. HUMIRA targets and helps block TNF-α, a specific source of inflammation that can have a role in uveitis. The FDA approval is based on results from two pivotal Phase 3 studies, VISUAL-I and VISUAL-II, which demonstrated that adult patients with active and controlled non-infectious intermediate, posterior and panuveitis treated with Humira had a significantly lower risk for treatment failure (a combination of uveitic flare and decrease in visual acuity), compared to placebo.
Here are a few other recent bits of regulatory and approval news:
ONT-380 gets Fast Track Designation for advanced HER2+ metastatic breast cancer
SEATTLE—Late June saw Cascadian Therapeutics, a clinical-stage biopharmaceutical company, announce that the ONT-380 program in advanced HER2+ metastatic breast cancer has received Fast Track designation from the FDA.
The company is actively recruiting patients for a randomized, double-blind, placebo-controlled Phase 2 study, known as HER2CLIMB, evaluating ONT-380 in combination with trastuzumab and capecitabine for patients with advanced HER2+ metastatic breast cancer. This trial is expected to enroll up to 180 patients with and without brain metastases. ONT-380 is an oral, highly selective small-molecule inhibitor of HER2.
“Fast Track designation recognizes the unmet medical need for this serious disease,” said Scott Myers, president and CEO of Cascadian Therapeutics. “Many patients with metastatic HER2+ breast cancer will see their disease progress despite the availability and use of multiple targeted therapies. We are encouraged by the early evidence of systemic activity and activity against brain metastases, and the favorable tolerability profile with ONT-380 in combination studies.”

FDA approves Gilead’s Epclusa for all genotypes of chronic hepatitis C
FOSTER CITY, Calif.—In late June, Gilead Sciences Inc. announced that the FDA had approved Epclusa (sofosbuvir 400 mg/velpatasvir 100 mg), the first all-oral, pan-genotypic, single-tablet regimen for the treatment of adults with genotype 1-6 chronic hepatitis C virus (HCV) infection. Epclusa is also the first single-tablet regimen approved for the treatment of patients with HCV genotype 2 and 3, without the need for ribavirin. Epclusa for 12 weeks was approved in patients without cirrhosis or with compensated cirrhosis (Child-Pugh A), and in combination with ribavirin for patients with decompensated cirrhosis (Child-Pugh B or C).
“The approval of Epclusa represents an important step forward in the global effort to control and potentially eliminate HCV, as it provides a safe, simple and effective cure for the majority of HCV-infected patients, regardless of genotype,” said Dr. Ira Jacobson, chairman of the Department of Medicine at Mount Sinai Beth Israel and a principal investigator in the Epclusa clinical trials. “Building on the established backbone of sofosbuvir, Epclusa demonstrated consistently high cure rates across all genotypes, including among patients with genotype 2 and 3, who traditionally have required ribavirin or other multi-pill regimens.”
Epclusa’s approval is supported by data from four international Phase 3 studies: ASTRAL-1, ASTRAL-2, ASTRAL-3 and ASTRAL-4.
“[This] approval represents a significant advance for patients with HCV genotypes 2 and 3, who previously required more complex and costly regimens,” said Dr. John Milligan, president and CEO of Gilead. “As the first and only pan-genotypic cure for hepatitis C, Epclusa has the potential to eliminate the need for genotype testing, which can be a barrier to treatment in certain resource-constrained settings. We look forward to making Epclusa available to patients around the world as quickly as possible.”

European Commission approves Opdivo+ Yervoy regimen for advanced melanoma
PRINCETON, N.J.—Bristol-Myers Squibb recently announced that the EC had approved Opdivo in combination with Yervoy for the treatment of advanced (unresectable or metastatic) melanoma in adults, representing the first and only approved combination of two immuno-oncology agents in the EU. Approval was based on CheckMate-067, a Phase 3 study in which the Opdivo+Yervoy regimen and Opdivo monotherapy demonstrated superior progression-free survival and objective response rates in patients with advanced melanoma, regardless of BRAF mutational status, compared to Yervoy alone.
“Historically, advanced melanoma has been a very difficult-to-treat disease,” said Dr. James Larkin of The Royal Marsden and lead author on CheckMate-067. “Now, with this approval, patients in Europe will have a treatment option combining two immuno-oncology therapies ... This is truly good news for healthcare providers and the patients they treat, as it represents an important new treatment option with the potential for improved outcomes.”

Atara Bio receives Advanced Therapy Medicinal Product classification
SOUTH SAN FRANCISCO—Atara Biotherapeutics Inc., a biopharmaceutical company focused on developing therapies for patients with severe and life-threatening diseases, recently announced that the European Medicines Agency has classified the company’s Epstein-Barr virus-specific cytotoxic T-lymphocyte (EBV-CTL) product candidate as an Advanced Therapy Medicinal Product that falls within the definition of a somatic cell therapy medicinal product. Atara’s EBV-CTLs are under development for the treatment of patients with EBV post-transplant lymphoproliferative disorder (EBV-PTLD).

FDA approves PaxVax’s single-dose oral cholera vaccine
REDWOOD CITY, Calif.—In June, PaxVax announced that it has received marketing approval from the FDA for Vaxchora, a single-dose oral, live attenuated cholera vaccine indicated for use in adults 18 to 64 years of age. Vaxchora is the only vaccine available in the United States for protection against cholera and the only single-dose vaccine for cholera currently licensed anywhere in the world.
“FDA approval of a new vaccine for a disease for which there has been no vaccine available is an extremely rare event. The approval of Vaxchora is an important milestone for PaxVax, and we are proud to provide the only vaccine against cholera available in the U.S.,” said Nima Farzan, CEO and president of PaxVax.
“As more U.S. residents travel globally, there is greater risk of exposure to diseases like cholera,” added Dr. Lisa Danzig, the company’s vice president of clinical development and medical affairs. “Cholera is an underestimated disease that is found in many popular global travel destinations and is thought to be underreported in travelers. Preventative measures such as food and water precautions can be challenging to follow effectively, and until now, U.S. travelers have not had access to a vaccine to help protect against this potentially deadly pathogen.”

Jeffrey Bouley

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