Rare disease drugs lead CHMP approvals

The latest crop of position opinions from the EU’s Committee for Medicinal Products for Human Use (CHMP) includes two drugs for rare diseases and two cancer therapies.

The rare disease therapies include two enzyme replacement therapies for genetic diseases that will be sold by Alexion, namely Kanuma (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency and Strensiq (asfotase alfa), the first therapy for the bone disease hypophosphatasia.

The marketing application for Kanuma was actually filed by Synageva BioPharma, a company which Alexion acquired last month in an $8.4 billion deal that is designed to bolster its rare-disease portfolio and reduce its reliance on $2 billion-selling blockbuster Soliris (eculizumab).

Analysts have suggested Kanuma could eventually become a $1.5 billion-a-year drug, while Strensiq has been tipped to make in excess of $1 billion.

Novartis got the nod from the CHMP for two cancer drugs – Farydak (panobinostat) for multiple myeloma and Odomzo (sonidegib) for the skin cancer basal cell carcinoma. Farydak is the first drug in the histone deacetylase (HDAC) inhibitor class to be backed by the EU panel as a third-line therapy for myeloma after earlier treatment with Takeda/Millennium’s Velcade (bortezomib) and an immuno-modulating agent.

The drug was approved in the US in February, and was the fourth HDAC-targeting drug to reach the market there after Merck & Co’s Zolinza (vorinostat), Celgene’s Istodax (romidepsin) and Spectrum Pharmaceuticals’ Beleodaq (belinostat). Merck withdrew its EU marketing application for Zolinza in 2009 and the CHMP rejected Celgene’s Istodax application in 2012, although Beleodaq remains in the pipeline in Europe.

Farydak launches into an increasingly competitive market in myeloma, currently dominated by Velcade, Amgen’s Kyprolis (carfilzomib) and Celgene’s Pomalyst (pomalidomide) and Revlimid (lenalidomide).

The positive opinion for Odomzo in basal cell carcinoma puts Novartis on course to compete in this common type of skin cancer against Roche’s Erivedge (vismodegib), which was approved in the US in 2012 and given conditional approval in Europe a year later. Roche’s drug made $128 million in sales last year, with the EU driving growth.

Other decisions

The CHMP also backed approval of CSL Behring’s Respreeza (human alpha1-proteinase inhibitor) for the treatment of the hereditary disease alpha1-proteinase inhibitor deficiency (AATD), which leads to chronic respiratory symptoms such as emphysema, but rejected CytoCure’s Heparesc, a treatment based on liver cells for urea cycle disorders.

The panel also gave its blessing to generic medicines, namely Hospira’s chemotherapy drug docetaxel, a copy of Lilly’s antidepressant Cymbalta (duloxetine) from Zentiva and Accord’s pregabalin, which is equivalent to Pfizer’s epilepsy brand Lyrica.

There were also green lights for two ‘hybrid’ medicines which combine preclinical tests and clinical data from the reference product with new data.

Novartis’ Sandoz unit got the nod for a generic version of Bristol-Myers Squibb/Otsuka’s big-selling antipsychotic drug Abilify (aripiprazole), while Santhera was given the go-ahead for Raxone (idebenone) for the treatment of visual impairment in patients with Leber’s hereditary optic neuropathy (LHON). Idebenone was previously sold by Takeda as Mnesis in various countries – including Italy – for cognitive disorders.

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