Four new molecular entities and an orphan medicine were granted positive opinions by the European Medicines Agency's scientific advisory panel, the Committee for Medicinal Products for Human Use (CHMP), during its meeting on July 18-21. Two of the NMEs, abiraterone and telaprevir, were submitted by the Johnson & Johnson subsidiary Janssen-Cilag International NV.

CHMP said it had assessed the marketing application for Janssen-Cilag's Zytiga (abiraterone acetate) 250mg tablets under an accelerated timetable because the product has a novel mechanism of action and because of the potential it has as an alternative therapeutic option for patients with prostate cancer.

Abiraterone is a once-daily oral androgen biosynthesis inhibitor and was recommended for use in combination with prednisone or prednisolone for metastatic castration-resistant prostate cancer in adult men whose disease has progressed on or after a docetaxel-based chemotherapy regimen.

A marketing approval for Zytiga was granted in the U.S. in April, heralding a battle for supremacy between a number of medicines recently approved for prostate cancer, including Sanofi SA's Jevtana (cabazitaxel) and Dendreon Corp.'s Provenge Also see "Zytiga Cleared Fast In Refractory CRPC, Can It Also Move To Earlier Use?" - Pink Sheet, 2 May, 2011.).

A second new medicine from Janssen-Cilag, the direct acting hepatitis C antiviral Incivo (telaprevir) was recommended for approval for the treatment of genotype 1 chronic hepatitis C in adult patients with compensated liver disease (see related story, Also see "J&J vs. Merck: The Race Is On As Incivek Passes First EU Regulatory Hurdle" - Pink Sheet, 22 July, 2011.).

ICU Sedative Cleared For Finland's Orion

Orion Corp.'s Dexdor (dexmedetomidine) has been marketed in the U.S. since 2000 by a licensee, Hospira Inc., as Precedex and has now received a positive opinion in the EU for use as an intensive care unit sedative. The compound is a relatively selective alpha 2-agonist, and is expected to be indicated for patients who do not require deep sedation. It reduces the time for extubation compared with the standard of care, CHMP noted.

A spokesman for the Finnish company told "The Pink Sheet" DAILY that Dexdor's development in Europe was behind that in the U.S. because earlier clinical studies did not satisfy the European regulatory authorities. Orion had to conduct two new Phase III trials, comparing it with two other sedatives, propofol and midazolam, which took some time.

First Rare Disease Drug From Pfizer

Pfizer Inc.'s Vyndaqel (tafamidis) 20 mg capsules is the first pharmacological product recommended for the treatment of transthyretin amyloidosis in adult patients with symptomatic polyneuropathy, a severe, progressive orphan disease. It is also the first product to emerge from Pfizer's new rare diseases unit, which was set up a year ago and was boosted with the acquisition of the U.S. biotech company FoldRx Pharmaceuticals, which was developing tafamidis in September 2010 Also see "Pfizer Adds To Specialty Care Division With Purchase Of FoldRx" - Pink Sheet, 1 September, 2010.). However, Pfizer received a setback in April 2011 when FDA issued a refusal to file letter for tafamidis, saying the application was incomplete Also see "FDA Sends Back Filing For Pfizer's Tafamidis, Acquired in FoldRx Deal" - Pink Sheet, 4 April, 2011.).

CHMP said it was aware that Pfizer was unable to provide comprehensive evidence on safety and efficacy; because of the rarity of the disease being treated, and moved to recommend approval under the EU's "exceptional circumstances" provision.

After 18 months therapy in the pivotal study, more patients treated with Vyndaqel met the definition of response with respect to the Neuropathy Impairment Score of the Lower Limb than patients on placebo, CHMP noted. Less deterioration of neurologic function, as measured by other endpoints and improved nutritional status, were also observed in patients treated with Vyndaqel.

Chronotherapy For Addison's

DuoCort Pharma AB's Plenadren (hydrocortisone) 5 mg and 20 mg modified-release tablets, were recommended for approval for the treatment of adrenal insufficiency in adults following the submission of a "hybrid" marketing application.

A hybrid application is one based on studies from the scientific literature, backed up by a small number of DuoCort's own clinical studies, and can be used for orphan products. Plenadren is a replacement therapy used in patients unable to produce their own cortisol. It has been designed to mimic the physiological cortisone profile seen in patients with adrenal insufficiency, also known as Addison's disease. It also has orphan drug status in the U.S.

New Indications For Afinitor, Enbrel And Tarceva

Additional indications recommended for approval by CHMP at its July meeting included: use for the treatment of neuroendocrine tumors of pancreatic origin, for Novartis SA's Afinitor (everolimus); extension to lower age limits, for Pfizer's Enbrel (etanercept); and use in first-line treatment of locally advanced or metastatic non-small cell lung cancer with EGF-activating mutations, for Roche Holdings' Tarceva (erlotinib).

However, the indications for Eli Lilly's Cymbalta (duloxetine) were not extended to include the treatment of moderate to severe chronic somatic pain in patients not taking NSAIDs regularly. CHMP said clinical studies had not proved the efficacy of duloxetine in the indication and there was insufficient evidence that its effect could be maintained over time. More information was also needed on the effects of duloxetine in the elderly.

A suspension formulation of the anticancer agent mercaptopurine from Nova Laboratories Ltd was recommended for approval in children as well as in adults and adolescents with acute lymphoblastic leukemia. It is the second positive opinion from CHMP under its pediatric-use marketing authorization program. Nova was established after a management buyout from the U.K. National Health Service in 1994 and supplies compounded products to hospitals and community pharmacies.

- John Davis (j.davis@elsevier.com)

Positive Opinions in Europe For J&J's Zytiga And Telaprevir, Pfizer's Tafamidis And Orion's Dexmedetomidine

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