On April 28, 2016 Medical Need part of Immedica Group reported that it has entered into an exclusive supply and distribution agreement with the French company Laboratoires CTRS, regarding marketing and sale of CTRS portfolio of pharmaceuticals in the Nordic region (Press release, Immedica Pharma, APR 28, 2016, View Source [SID1234555256]). In 2013, CTRS received EU marketing authorization and orphan drug market exclusivity for its product Orphacol (cholic acid), indicated for the treatment of two rare inborn errors of metabolism in the primary bile acid synthesis: 3β-hydroxy-Δ5-C27-steroid oxidoreductase and Δ4-3-oxosteroid-5β-reductase deficiency. A few weeks ago, the European commission approved a centralized marketing authorization for the company’s second product, Neofordex, containing a high and appropriate dosage (40 mg) of dexamethasone, a common component used in combination with other pharmaceuticals in the treatment of multiple myeloma. While already well established in the treatment protocols, prior to the approval of Neofordex, dexamethasone has only been available in low strengths (0.5-4 mg), forcing patients to take a very high number of tablets (10-80 per day) to achieve an appropriate dosage. In addition to the increased convenience for the patients, since an adequate dose exposure is critical for efficacy of the treatment regimen, the expectation is that the availability of a tablet in appropriate strength could improve compliance and thereby potentially the treatment outcome. Under the agreement, Medical Need gains the rights to Neofordex and Orphacol in Denmark, Finland, Iceland, Norway and Sweden, and will be responsible for the distribution, marketing and sale of the products in that territory. "CTRS has a very exciting portfolio which fits well with our competencies and capabilities in Medical Need", said Tomas Gloveus, Head of Marketing and Sales at Medical Need, and continued, "Orphacol and Neofordex both fulfil high unmet medical needs, which have previously not been adequately served in this region, and we look forward to now being able to make these products available to the affected patients in the Nordic countries."
About Multiple Myeloma and Neofordex
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Multiple myeloma (MM) is a form of cancer that affects a type of white blood cells called plasma cells. Plasma cells are part of the immune system and normally helps the body fight infections by producing antibodies. Rather than producing helpful antibodies, in MM, the cancer cells produce abnormal proteins that over time damages the kidneys. MM also causes cancer cells to accumulate in the bone marrow, where they crowd out healthy blood forming cells which results in a deficiency of red and white blood cells, as well as blood platelets. This in turn leads to a number of different symptoms, depending on the deficient cell; anemia (paleness, fatigue), leukopenia (sensitivity to infection) and thrombocyotopenia (bleeding and bruising). MM also causes damage to the bone and skeleton. MM is slightly more prevalent in men and typically affects the elderly (>65), but may affect individuals as young as 30. MM is treated by a number of treatment protocols, which typically involve several different pharmaceuticals. Over the past years, several new products have been approved for the treatment of MM as part of such treatment protocols. A common component in most of the treatment protocols is dexamethasone (denoted as "d" or "D" in the protocol abbreviations), a potent and long-acting corticosteroid, which has been shown to play an important part in the efficacy of the different regimens. The typical daily dose of dexamethasone in the treatment of MM is 40 mg. Neofordex is the only approved pharmaceutical which contains 40 mg of dexamethasone in a single tablet. Prior to the approval of Neofordex, dexamethasone has only been available in low strengths (0.5-4 mg), forcing patients to take a very high number of tablets (10-80 per day) to achieve an appropriate dosage. An adequate dose exposure of dexamethasone has been shown to be of high importance for the efficacy of the treatment regimen (Kobayashi et al., Int J Hematol. 2010 Nov;92(4):579-86). It is the expectation that the availability of a tablet in an appropriate strength, in addition to the improved convenience from reducing the pill burden for the patients, could translate into increased compliance and thereby potentially improving the treatment outcome. Neofordex was on 2016-03-16 granted a centralized marketing authorization by the European Commission, valid for all markets of the EU, Norway and Iceland. Prior to the regulatory approval, the product has been used extensively on a named patient basis in France, under what is called an ATU cohort, including most French MM patients. The product is planned to be launched in the Nordic and Baltic markets during Q2 2016.
About Inborn Errors of Primary Bile Acid Synthesis and Orphacol
Inborn errors of primary bile acid synthesis are very rare inherited conditions, caused by mutations in the genes encoding certain enzymes responsible for the liver’s production of bile acids, one of the key components of the bile. Left untreated, these enzyme deficiencies lead to the accumulation of hepatotoxic metabolites and progression to irreversible cholestasis and liver failure, and are usually fatal. Orphacol is the only approved treatment for two particular inborn errors in the primary bile acid synthesis: 3β-hydroxy-Δ5-C27-steroid oxidoreductase and Δ4-3-oxosteroid-5β-reductase deficiency. Orphacol contains cholic acid, which acts through a dual mechanism, by suppressing the faulty bile acid synthesis thus reducing formation of hepatotoxic metabolites and by restoring the biliary secretion and elimination of toxic metabolites through the bile. It also corrects the intestinal malabsorption of fats and fat-soluble vitamins, thereby improving the child’s growth. Treating affected patients with Orphacol can avoid the need for a liver transplantation, an operation with very serious potential consequences, especially in young children. Orphacol was granted a centralized marketing authorization by the European Commission, valid for all markets of the EU, Norway and Iceland in 2013. The product also enjoys orphan drug market exclusivity for 10 years following marketing authorization. The product will immediately be made available on the Nordic and Baltic markets.