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A collection of recent regulatory approvals and actions globally

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From orphan drug designation for a gene therapy that treats mucopolysaccharidosis type I to approval of a hemophilia treatment, we have a number of quick items to present to you and give you an idea of the breadth and depth of recent regulatory activity in Europe and the United States.

RGX-111 gene therapy gets orphan drug status
ROCKVILLE, Md.—Early October saw REGENXBIO Inc., a biotechnology company involved in gene therapy, announce that the U.S. Food and Drug Administration (FDA) had granted its Orphan Drug Designation to the company’s investigational gene therapy product candidate RGX-111 for the treatment of mucopolysaccharidosis type I (MPS I).
“REGENXBIO is pleased to have received Orphan Drug Designation from the FDA for RGX-111,” said Kenneth T. Mills, president and CEO of REGENXBIO. “MPS I is a severely debilitating disease, and patients and their caregivers do not currently have adequate therapeutic options. We remain committed to our vision of developing gene therapies for patients with high unmet medical needs, including MPS I.”
Orphan drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.
MPS I is a rare neurodegenerative disease caused by deficiency of the a-l-iduronidase (IDUA) gene, and somewhere around 1,000 individuals with MPS I are estimated to be born each year worldwide. Symptoms include excessive accumulation of fluid in the brain, spinal cord compression and cognitive impairment. RGX-111 uses an AAV9 vector to deliver the IDUA gene to the central nervous system.
REGENXBIO intends to file an Investigational New Drug Application (IND) with the FDA in the first half of 2016 to support the initiation of a dose-escalation Phase 1/2 clinical trial of RGX-111 beginning in the first half of 2016.

EMA fast-tracks antidote to anticoagulant Pradaxa
INGELHEIM, Germany—The European Medicines Agency (EMA) recently recommended granting a marketing authorization, following accelerated assessment, for Boehringer Ingelheim’s Praxbind (idarucizumab) as a specific antidote to the anticoagulant medicine Pradaxa (dabigatran etexilate), when rapid reversal of its effect is required. Praxbind is to be used when a patient taking Pradaxa needs to undergo an emergency surgery or when life-threatening or uncontrolled bleeding occurs.
Pradaxa belongs to a new generation of oral anticoagulants approved over the past few years, which have given doctors and patients a wider range of options to prevent and treat thromboembolic disorders in adults. Praxbind is the first medicine designed to specifically neutralize the anticoagulant effect of Pradaxa.
Bleeding is a well-known complication of all anticoagulants, and information on how to address this risk has been included in Pradaxa’s product information since it was first authorized in the European Union (EU) in March 2008. Although low in frequency in patients treated with Pradaxa, major and sometimes life-threatening bleeding may occur. However, unlike older oral anticoagulants such as warfarin, up until now there has been no specific means of rapidly neutralizing Pradaxa’s effect.

New medicine to treat heart failure recommended for approval
BASEL, Switzerland—The EMA’s Committee for Medicinal Products for Human Use recently recommended granting a marketing authorization for Novartis’ Entresto (sacubitril/valsartan) for the treatment of adults with symptomatic chronic heart failure with reduced ejection fraction, a condition where the heart muscle does not contract effectively and less oxygen-rich blood is pumped out to the body. Around half of people with heart failure will have reduced ejection fraction.
Entresto is a combination of valsartan (an angiotensin receptor blocker, or ARB) and sacubitril. Sacubitril is the first in a new class of medicines called neprilysin inhibitors. Entresto works in two ways—valsartan blocks the angiotensin II type-1 receptor, suppressing the harmful effects of angiotensin II on the cardiovascular system, while sacubitril blocks an enzyme known as neprilysin to enhance the protective neurohormonal systems of the heart. Because of its mechanism of action Entresto should not be given together with another ARB or with an angiotensin converting enzyme (ACE) inhibitor.
The efficacy of Entresto compared with enalapril (an ACE inhibitor) was assessed in one randomized controlled trial including over 8,000 adults with heart failure with reduced ejection fraction. Patients also received other heart-failure medicines. The trial was stopped early when it was found that Entresto was more effective than enalapril in reducing deaths from cardiovascular disease.

Zalviso gains marketing authorization in EU
REDWOOD CITY, Calif.—AcelRx Pharmaceuticals Inc. has announced that the European Commission (EC) approved Zalviso (15 micrograms sufentanil sublingual tablets) for the management of acute moderate-to-severe postoperative pain in adult patients. The marketing authorization is granted for the 28 EU member states as well as for the European Economic Area (EEA) countries of Norway, Iceland and Liechtenstein. Zalviso is a system combining a drug and a device designed to deliver a sublingual tablet formulation of sufentanil 15 mcg via a proprietary, preprogrammed, noninvasive, patient-controlled analgesia device. Grunenthal Group, AcelRx’s licensee in Europe and Australia, expects the product to be available to Western European patients in the first half of 2016.
“This is a significant event for AcelRx. Not only is this the company’s first marketing approval, but it represents the successful development and commercialization of a product that we believe will provide a new way for physicians and their patients to treat acute moderate-to-severe postoperative pain using an innovative delivery method,” stated Howie Rosen, interim CEO of AcelRx Pharmaceuticals. “Our partner Grunenthal will be working with the member states of the EU and EEA to ensure that Zalviso is made available to those patients who would benefit from an effective and reliable solution for their moderate-to-severe postsurgical pain.”

Taiho Oncology announces FDA approval of Lonsurf
PRINCETON, N.J.—Taiho Oncology Inc. (U.S.), a subsidiary of Taiho Pharmaceutical Co. Ltd. in Japan, announced in late September that the FDA had approved Lonsurf (trifluridine and tipiracil), formerly known as TAS-102, for the treatment of patients with metastatic colorectal cancer who have been previously treated with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy, an anti-VEGF biological therapy and, if RAS wild-type, an anti-EGFR therapy.
“Patients with metastatic colorectal cancer, whose disease has progressed after treatment with standard therapies, have had limited therapeutic options to treat their disease,” said Eric Benn, Taiho Oncology’s president and CEO. “Lonsurf helps address this unmet medical need by providing patients with a new therapeutic option that can help extend their overall survival. As the first FDA approval for Taiho Oncology, Lonsurf also represents a major milestone for our company.”
“Metastatic colorectal cancer cells often become resistant to previously effective treatment, underscoring the importance of identifying new therapeutic options for patients with the disease,” said Dr. Robert J. Mayer, faculty vice president for academic affairs at the Dana Farber Cancer Institute, professor of medicine at Harvard Medical School, and principal investigator of the RECOURSE trial that studied the compound. “In a pivotal clinical trial, Lonsurf demonstrated that it can extend overall survival, providing patients and their oncologists with a novel oral therapy.”
Lonsurf was approved in Japan in March 2014 and is indicated to treat unresectable advanced or recurrent colorectal cancer.

FDA grants Fast Track Designation to MDX for Fragile X Syndrome
TEL AVIV, Israel—Alcobra Ltd., an emerging pharmaceutical company focused on the development of new medications to help patients with cognitive disorders, including attention deficit hyperactivity disorder (ADHD) and fragile X syndrome, announced recently that the FDA had granted Fast Track designation to Metadoxine Extended Release (MDX) for the treatment of fragile X syndrome.
“We are pleased that the FDA has recognized the potential of MDX in fragile X syndrome and granted Fast Track designation for this indication,” said Dr. Yaron Daniely, president and CEO of Alcobra. “We look forward to our upcoming meeting with the FDA to determine next steps in advancing the development program for MDX in this area of serious unmet medical need.”
Companies that receive Fast Track designation can have more frequent interactions with the FDA review team to facilitate product development. In addition, based on clinical data, the NDA applications for these products could be eligible for priority and/or rolling review.
In 2013, the FDA granted orphan status to metadoxine for the treatment of fragile X syndrome, a condition for which there are currently no FDA-approved medications.

QIDP status goes to Taksta antibiotic
CHAPEL HILL, N.C.—Mid-September saw Cempra Inc., a clinical-stage pharmaceutical company focused on developing antibiotics to meet critical medical needs in the treatment of bacterial infectious diseases, announce that the FDA had granted a qualified infectious disease product (QIDP) designation to Cempra’s investigational antibiotic product candidate, Taksta (CEM-102, sodium fusidate, the sodium salt of fusidic acid). The designation is for Taksta oral tablets for the indication of acute bacterial skin and skin structure infections (ABSSSI).
“Taksta is Cempra’s second antibiotic product candidate to obtain QIDP status, which should enable us to expedite its development and bring this promising drug to the patients who need it the most,” said Dr. Prabhavathi Fernandes, president and CEO of Cempra. “Previously our lead product, solithromycin, received QIDP status, and together we view these designations as further validation of Cempra’s progress in achieving its goal of becoming a leader in the global anti-infective market.”
The QIDP designation was created by the Generating Antibiotic Incentives Now (GAIN) Act of 2012. It provides certain incentives for the development of new anti-infectives, including eligibility for priority review, the FDA’s Fast Track program and a five-year extension of exclusivity under the Hatch-Waxman Act.
Cempra is developing Taksta exclusively in the United States for ABSSSI and is exploring its use for the long-term oral treatment of refractory bone and joint infections, including prosthetic joint infections. Fusidic acid is orally active against gram-positive bacteria, including all Staphylococcus aureus strains, such as HA-MRSA and CA-MRSA.

FDA approves Octapharma’s Nuwiq for hemophilia A
HOBOKEN, N.J.—Octapharma USA announced Sept. 15 that the FDA had approved Nuwiq, Antihemophilic Factor (Recombinant), an intravenous therapy for adults and children living with hemophilia A. The Nuwiq approval includes on-demand treatment and control of bleeding episodes, routine prophylaxis to reduce the frequency of bleeding episodes and perioperative management of bleeding.
Nuwiq is the first B-domain deleted recombinant factor VIII (FVIII) derived from a human cell line, not chemically modified or fused with another protein, designed for the treatment of patients with hemophilia A, congenital FVIII deficiency. Although present therapies for hemophilia A treatment exist in the United States, significant challenges still remain, including development of inhibitors and the need for multiple infusions on a prophylactic basis.
“Octapharma has been committed to the bleeding disorders community for many years, and its decade-long drive to find solutions for hemophilia A challenges has never wavered,” said Octapharma USA President Flemming Nielsen.

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