Eight medicines have taken a big leap towards being approved in Europe having won the support of the European Medicines Agency’s Committee for Medicinal Products for Human Use.
First up, approval of Intercept Pharma’s orphan drug Ocaliva (obeticholic acid) is being backed for the treatment of the rare, chronic and life-threatening liver disease primary biliary cholangitis (also known as primary bile cirrhosis).
Aside from liver transplantation, ursodeoxycholic acid (UDCA) is the only medicine currently available to treat the condition, but up to half of all patients given the drug either fail to respond to it or experience limited benefits, highlighting the need for new treatment options.
It is hoped that Ocaliva could help address this unmet need. The drug is a semi-synthetic bile acid that works by activating the farnesoid X receptor (FXR), which controls the production of bile, thereby potentially reducing the exposure of the liver to toxic levels of bile acids.
The Committee also recommended granting a conditional marketing authorisation for AbbVie’s orphan drug Venclyxto (venetoclax) to treat adults with chronic lymphocytic leukaemia (CLL).
According to the regulator, single-arm studies with Venclyxto have shown its ability to produce responses in patients unsuitable for or refractory to B-cell receptor pathway inhibitors and other anticancer medicines.
The drug is currently available to UK patients through the country’s Early Access to Medicines Scheme, which enables patients with life-threatening and seriously debilitating conditions to receive innovative and promising new drugs as soon as the MHRA has signalled that the benefits outweigh the risks.
Use of Orphan Europe’s Cystadrops (mercaptamine) has been endorsed by the CHMP for the treatment of corneal cystine crystal deposits in patients with cystinosis.
Cystinosis is a rare genetic metabolic disease that causes build up of the amino acid cystine in various organs of the body. Cystadrops, which also carries orphan status, can reduce accumulation in the cornea.
Ferring Pharmaceuticals’ Rekovelle (follitropin delta) was recommended for approval for controlled ovarian stimulation in women undergoing assisted reproductive technologies. The treatment can stimulate the development of multiple mature follicles in the ovaries.
The Committee recommended granting a marketing authorisation for the Advanced Accelerator Applications’ orphan therapy SomaKit-TOC (edotreotide) for the diagnosis of gastro-entero-pancreatic neuroendrocrine tumours.
The active substance edotreotide facilitates radiolabelling with gallium (68Ga) chloride solution and binds with high affinity to somatostatin receptors (SSTRs) in tumours that overexpress them.
The CHMP also put three generic medicines were forward for approval, all intended to treat HIV infection in adults: Emtricitabine/Tenofovir disoproxil Mylan (emtricitabine/tenofovir disoproxil); Emtricitabine/Tenofovir disoproxil Krka (emtricitabine/tenofovir disoproxil); and Tenofovir disoproxil Mylan (tenofovir disoproxil).
