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A collection of recent regulatory approvals and other actions globally

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We begin this regulatory roundup with end-of-September news that the U.S. Food and Drug Administration (FDA) approved Stelara (ustekinumab), a biologic therapy from Janssen Biotech Inc., for the treatment of moderately to severely active Crohn’s disease in adults—making it the first biologic therapy to target interleukin-12 and interleukin-23 cytokines for the treatment of Crohn’s disease.
Stelara will be available for use in adult patients with Crohn’s disease who have failed or were intolerant to treatment with immunomodulators or corticosteroids but never failed treatment with a tumor necrosis factor (TNF) blocker. It has also been approved for use in patients who failed or were intolerant to treatment with one or more TNF blockers.
“Because of the individual nature of these diseases, what works for one patient may not work for another. That is why it is so critical that our Crohn’s patients have many different treatment options available to them,” said Michael Osso, president and CEO of the Crohn’s & Colitis Foundation of America (CCFA), in an official statement about the FDA decision. “The approval of Stelara is extremely important for patients living with moderate-to-severe Crohn’s disease. Many of these patients have exhausted available treatments, and Stelara gives them another option to hopefully induce remission, help manage their disease, and improve their quality of life.”
“The role of cytokines in regulating the immune system’s response to inflammation has been the subject of significant research. Through our robust grant-funding program, CCFA has funded a number of preliminary research studies into the importance of cytokines in the treatment of IBD [irritable bowel disease],” Osso continued. “We will continue to fund research into cytokines and other potential therapeutic targets in order to yield additional discoveries and new treatments for IBD patients.”

FDA approves Solana influenza assay
NEW YORK—Late September also saw Quidel announce that it had received FDA 510(k) approval for its Solana assay for the diagnosis of influenza A and B. The test uses the helicase-dependent amplification technology that, the company says, “underpins Quidel’s AmpliVue line of molecular assays,” and is designed to detect nucleic acids isolated from nasal and nasopharyngeal swabs from patients with signs and symptoms of respiratory infection. It is intended for use only with the company’s Solana instrument.
“A single Solana instrument system performs up to 12 Solana Influenza A+B assays on almost any sample type or brand of viral transport media in under an hour, and up to 96 patient samples during an eight-hour shift, which is critical during an influenza epidemic when testing volumes are at their highest and samples are coming from several different locations and often collected in a variety of different transported media,” said Douglas Bryant, Quidel’s president and CEO.
The FDA approval came about a month after the agency cleared Quidel’s Solana assay for the sexually transmitted disease trichomoniasis, and a little more than a year after it approved the firm’s Solana group A streptococcus assay and the Solana platform itself.

AbbVie HCV regimen receives Breakthrough Therapy status
NORTH CHICAGO, Ill.—AbbVie, a global biopharmaceutical company, announced recently that the FDA granted Breakthrough Therapy designation for the investigational, pan-genotypic regimen of glecaprevir (ABT-493)/pibrentasvir (ABT-530) for the treatment of patients with chronic hepatitis C virus who failed previous therapy with direct-acting antivirals in genotype 1, including therapy with an NS5A inhibitor and/or protease inhibitor.
The designation is supported by positive results seen in AbbVie’s Phase 2 MAGELLAN-1 clinical study.
“AbbVie is committed to advancing HCV care and addressing areas of continued unmet need for people living with chronic HCV,” said Dr. Michael Severino, executive vice president of research and development and chief scientific officer of AbbVie. “The FDA’s Breakthrough Therapy Designation is an important step in our effort to bring our pan-genotypic regimen to market, which we are also investigating as an eight-week path to virologic cure for the majority of patients.”

EU authorization granted for Teva’s Aerivio Spiromax
JERUSALEM—Teva Pharmaceutical Industries Ltd. received EU Marketing Authorization for Aerivio Spiromax (fluticasone/salmeterol 500/50) this fall as a maintenance bronchodilator treatment for adult patients with severe asthma and chronic obstructive pulmonary disease (COPD). Aerivio Spiromax contains a fixed-dose combination of fluticasone propionate, an inhaled corticosteroid to treat the underlying inflammation in asthma and COPD and salmeterol xinafoate, a long-acting beta-agonist, being delivered via the Spiromax inhaler.
“We are excited about the European approval of Aerivio Spiromax that can give patients with severe asthma and COPD the possibility to benefit from this widely used fixed-dose combination in the award-winning Spiromax inhaler,” said Luca Frangoni, a Teva vice president and the head of the company’s respiratory efforts in Europe. “It is an important goal of our growing respiratory franchise to bring a wide range of new treatment options to patients and healthcare professionals, from innovative treatments and value-adding services to advanced inhalers such as Spiromax.”
With the approval of Aerivio Spiromax, Teva adds another established fixed-dose combination of inhaled corticosteroid with long-acting beta-agonist to its inhaler family. DuoResp Spiromax, the combination of budesonide and formoterol fumarate dihydrate, already received EU marketing authorization for the treatment of asthma and COPD in May 2014.

Rare Pediatric Disease nod for ezutromid
OXFORD, U.K.—In late September, Summit Therapeutics plc, a drug discovery and development company advancing therapies for Duchenne muscular dystrophy (DMD) and Clostridium difficile infection, announced it had received the Rare Pediatric Disease designation from the FDA for ezutromid in the treatment of DMD. As a utrophin modulator, ezutromid has potential as a disease-modifying treatment for all patients with the fatal muscle wasting disease DMD, regardless of their underlying dystrophin gene mutation.
“Rare Pediatric Disease designation builds upon the Fast Track and Orphan Drug designations which the FDA has already awarded to ezutromid, recognizing a significant unmet medical need in the treatment of DMD,” said Glyn Edwards, CEO of Summit. “We plan to leverage these regulatory advantages in the continued clinical development of ezutromid, which is currently in a Phase 2 clinical trial called PhaseOut DMD, to bring ezutromid to patients in need as quickly as possible.”

New treatment for patients with soft tissue sarcoma
LONDON—The European Medicines Agency (EMA) has recommended granting a conditional marketing authorization to Eli Lilly and Co.’s Lartruvo (olaratumab) for the treatment of adults with soft tissue sarcoma, a rare type of cancer. Lartruvo is to be used in combination with doxorubicin (a chemotherapy medicine) in patients with advanced soft tissue sarcoma for whom surgery or radiotherapy is not suitable, and who have not been previously treated with doxorubicin.
Lartruvo is a monoclonal antibody designed to recognize and attach to a protein called platelet-derived growth factor receptor alpha (PDGFRα). In soft tissue sarcoma, this protein is present in high levels or is overactive, causing cells to become cancerous. When Lartruvo attaches to PDGFRα on sarcoma cells, it blocks its activity, thereby slowing down the growth of the cancer.

Gene therapy accepted into PRIME Program
CAMBRIDGE, Mass.—Around the middle of September, bluebird bio Inc., a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, announced that the EMA had granted access to its Priority Medicines (PRIME) scheme for LentiGlobin drug product in the treatment of patients with transfusion-dependent beta-thalassemia.
The PRIME initiative provides enhanced support and increased interaction to companies, with the goal of optimizing development plans and speeding regulatory evaluations to potentially bring innovative medicines to patients more quickly. To be accepted for PRIME, a therapy must demonstrate potential to benefit patients with unmet medical need through early clinical data or nonclinical data. Access to the PRIME initiative complements bluebird’s ongoing participation in the EMA’s Adaptive Pathways Pilot program, which also aims to expedite patient access to therapies with the potential to treat serious conditions with unmet need. It uses the existing EU regulatory framework for medicines, including conditional approval.

Fast Track status for niraparib
WALTHAM, Mass.—TESARO Inc., an oncology-focused biopharmaceutical company, was recently granted Fast Track designation for niraparib for the treatment of patients with recurrent platinum-sensitive ovarian, fallopian tube or primary peritoneal cancer. TESARO has initiated a rolling submission of a New Drug Application for niraparib to the FDA and intends to complete this submission during the fourth quarter. The Marketing Authorization Application for niraparib is planned for submission to the EMA in the fourth quarter as well.

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