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A collection of recent regulatory approvals and actions globally

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Leading our roundup of regulatory actions on various therapeutics since December 2014 is Prezcobix, with Titusville, N.J.-based Janssen Therapeutics, a division of Janssen Products, announcing Jan. 29 that the U.S. Food and Drug Administration (FDA) has approved the compound, an HIV-1 protease inhibitor combined with a CYP3A4 inhibitor, for the treatment of human immunodeficiency virus (HIV-1) in combination with other antiretroviral agents for treatment-naïve and treatment-experienced adults with no darunavir resistance-associated substitutions.
Prezcobix is a once-daily, fixed-dose antiretroviral combination tablet containing 800 mg of darunavir, marketed as Prezista in the United States, and 150 mg of cobicistat, a pharmacokinetic enhancer or “boosting” agent, developed and marketed as Tybost by Gilead Sciences Inc., taken orally with other HIV-1 medications and with food.
“Additional options remain an important medical priority to meet the diverse needs of those living with and managing this disease,” said Dr. Karen Tashima, professor of medicine in the Division of Infectious Diseases of Brown University and director of HIV Clinical Studies, Miriam Hospital—she was also a lead investigator in the GS-US-216-0130 study for the compound. “This approval gives physicians the option of a darunavir-based fixed-dose combination tablet to treat adults living with the HIV-1 infection, which can help reduce the number of pills in their overall treatment regimen.”
The FDA approval was based on bioequivalence data evaluating the use of a darunavir and cobicistat fixed-dose combination tablet versus single agents (TMC114IFD1003) and a clinical study evaluating the safety and efficacy of cobicistat-boosted darunavir for the treatment of HIV-1 in adults with no darunavir resistance-associated mutations (GS-US-216-0130).
Here are some other notable recent approvals and actions by U.S. and European regulatory bodies:
First stem-cell therapy recommended for approval in EU
The European Medicines Agency (EMA) has recommended Holoclar from Chiesi Farmaceutici S.p.A., the first advanced therapy medicinal product containing stem cells, for approval in the European Union. Holoclar is a treatment for moderate-to-severe limbal stem cell deficiency (LSCD) due to physical or chemical burns to the eyes in adults. It is the first medicine recommended for LSCD, a rare eye condition that can result in blindness.  
The recommendation was made by the Committee for Medicinal Products for Human Use (CHMP) based on a robust assessment carried out by the Committee for Advanced Therapies, the Agency’s expert committee for ATMPs.  
“This recommendation represents a major step forward in delivering new and innovative medicines to patients,” said Enrica Alteri, head of EMA’s Human Medicines Evaluation Division. “EMA has used all available support tools to facilitate the development and assessment of Holoclar. It is an advanced therapy medicinal product that has been designated as an orphan medicine. This allowed the Agency to provide support, including several rounds of free scientific advice, to the applicant during Holoclar’s development.”
Holoclar is a living tissue equivalent intended to be transplanted in the affected eye(s) after removal of the altered corneal epithelium. It is made from a biopsy taken from a small undamaged area (minimum of 1 to 2 mm2) of the patient’s cornea and grown in the laboratory using cell culture. Holoclar can offer an alternative to transplantation for replacing altered corneal epithelium in some cases, and it has been shown to increase the chances of a successful corneal transplant where the injury has caused extensive eye damage. It reduces the risk of rejection compared with transplanting tissue from a donor and does not require surgery on the patient’s other eye, as only a small biopsy is performed to collect the cells, thus reducing the risk of damage to the healthy eye. Therefore, Holoclar may also be suitable where both eyes are affected by moderate-to-severe LSCD.
Novartis gains two FDA approvals
Novartis announced in late January that the FDA has granted accelerated approval of Bexsero (Meningococcal Group B Vaccine [recombinant, adsorbed]) for active immunization to prevent invasive meningococcal disease caused by serogroup B (also known as meningitis B) in adolescents and young adults from 10 years through 25 years of age. Bexsero is the only meningitis B vaccine approved in the United States with a two-dose regimen and a flexible dosing schedule. As part of the accelerated approval process, Novartis will complete its ongoing studies to confirm the effectiveness of Bexsero against diverse serogroup B strains.
“While rare, meningitis B is a devastating disease that can hit anyone anytime, especially teenagers and children,” said Andrin Oswald, division head of Novartis Vaccines. “This approval is an important milestone towards our goal of helping to prevent any further loss of life.”
Around the same time, Novartis also announced FDA approval for first IL-17A antagonist Cosentyx (secukinumab) for moderate-to-severe plaque psoriasis in adult patients who are candidates for systemic therapy or phototherapy. Cosentyx is the first approved psoriasis medication to selectively bind to IL-17A and inhibit its interaction with the IL-17 receptor. The approval is based on the efficacy and safety outcomes from 10 Phase 2 and 3 studies, including over 3,990 patients with moderate-to-severe plaque psoriasis, which demonstrated that Cosentyx resulted in clear or almost clear skin in the majority of patients and had an acceptable safety profile.
“We have heard first-hand from patients and doctors about the negative impact of psoriasis and the need for innovative therapies. With the FDA approval of Cosentyx, a majority of people living with moderate-to-severe plaque psoriasis in the U.S. now have available a new medication that can help them achieve clear or almost-clear skin,” said Christi Shaw, U.S. country head, president of Novartis Corp, and Novartis Pharmaceuticals Corp. “As the first approved treatment targeting the IL-17A pathway, which is believed to play a key role in the development of psoriasis, we are confident this novel treatment will make a difference for the psoriasis community.”
Mysimba recommended for approval in weight management
Mid-December saw a statement from the EMA for recommendation to approve a marketing authorization for Mysimba (naltrexone/bupropion) from Orexigen Therapeutics for weight management of overweight or obese adults. The medicine is recommended for use in addition to a reduced-calorie diet and physical activity.
Mysimba is a prolonged-release tablet to be taken orally. It is a combination of two active substances already approved for use in the EU in other indications. These active substances affect two key areas of the brain responsible for the control of food intake and energy expenditure, and for the reward pathways associated with eating food.
The effectiveness of Mysimba was assessed in four pivotal studies that included obese and overweight patients with and without weight-related conditions treated for one year. All patients enrolled in trials were required to change their lifestyle to incorporate a reduced-calorie diet and regular physical activity. Across these studies, more patients treated with Mysimba achieved clinically-relevant weight loss than patients treated with placebo.
The CHMP opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorization.
Rapivab approved for acute uncomplicated influenza  
BioCryst Pharmaceuticals Inc., a pharmaceutical company focused on the development and commercialization of treatments for rare and infectious diseases, announced Dec. 22 that the FDA had approved Rapivab (peramivir injection), an intravenous neuraminidase inhibitor, for the treatment of acute uncomplicated influenza in patients 18 years and older who have been symptomatic for no more than two days.
“RAPIVAB is the first neuraminidase inhibitor that has shown to be safe and effective as a single-dose, IV therapy for patients with acute, uncomplicated influenza, and represents the first new antiviral treatment for influenza approved by the FDA in 15 years,” said Dr. Richard Whitley of the University of Alabama at Birmingham. “In a blinded, randomized placebo-controlled trial, a single dose of Rapivab alleviated flu symptoms and reduced fever significantly faster than placebo.”
“The approval of Rapivab provides a new choice to immediately deliver an effective treatment in one dose to adult patients with influenza,” said Jon P. Stonehouse, president and CEO of BioCryst. “This is the first U.S. approval of a BioCryst-discovered drug and represents an important milestone for our company.”

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