Actelion’s Uptravi and rare bleeding disorder drug get positive EMA decisions
pharmafile | February 1, 2016 | News story | Sales and Marketing |Â Â CHMP, EMA, Uptravi, pulmonary arterial hypertension, selexipagÂ
The Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the EMA, has issued positive opinions on drugs from Actelion and Bio Products Laboratory, taking them a step closer to full EU approval.
Uptravi
Actelion’s Uptravi (selexipag) is a selective IP prostacyclin receptor agonist used to treat pulmonary arterial hypertension (PAH). Efficacy has been shown in a PAH population including idiopathic and heritable PAH, PAH associated with connective tissue disorders, and PAH associated with corrected simple congenital heart disease.
PAH is a form of high blood pressure affecting the arteries in the lungs and the right side of the heart. The illness is progressive and can sometimes be fatal.
The CHMP’s positive opinion is in part based on long-term data from the Phase III GRIPHON trial, a long-term Phase III study in PAH patients with WHO Functional Class I-IV symptoms.
Actelion chief executive Jean-Paul Clozel says: “We are delighted by today’s announcement of a positive CHMP opinion. Coming hot on the heels of both the US and Canadian approvals, we believe that Uptravi can significantly improve long-term outcomes for PAH patients. Once market authorisation is granted by the EU Commission, Uptravi will open up the prostacyclin pathway to many more patients.”
The European Commission is expected to issue a final decision on Uptravi by early April 2016. Swiss-based Actelion already started selling the potential blockbuster drug in the US last month.
Coagadex
Meanwhile, the CHMP also recommended approval of Coagadex (human coagulation factor X): a potential breakthrough drug for a rare bleeding disorder, manufactured by UK-based Bio Products Laboratory.
Coagadex is indicated for the treatment and preventive management of bleeding episodes and the control of bleeding during surgical procedures in patients with hereditary factor X deficiency. The medicine’s active substance is human coagulation factor X, a protein derived from human plasma.
Factor X is necessary for the blood coagulation (clotting) process. Patients with this condition lack sufficient levels of the protein and are therefore more prone to bleeding than other people and show prolonged bleeding after injury or surgery. Bleeding can also occur within muscles or the spaces in the joints, which can lead to permanent injury if it happens repeatedly.
Current treatment for this condition includes replacement therapies, which contain a mix of coagulation factors; however, these treatments are associated with problems with dosing and the risk of elevating other clotting factors which may result in complications. Due to the lack of specific treatment options, CHMP decided to speed up the evaluation of this medicine and recommended marketing authorisation following an accelerated assessment.
The committee based its recommendation on the results of trials, which were notably small in participant numbers due to the rarity of the condition. A Phase III study treated 207 spontaneous bleeds in 16 patients with moderate or severe factor X deficiency and demonstrated an effective response – excellent or good in 98.8% of the bleeds treated. Five patients participating in the study underwent seven surgical procedures and Coagadex was also shown to be effective for the prevention of bleeding during planned surgery. An additional study in children with mild to severe hereditary factor X deficiency is ongoing.
Despite the limited safety information available due to the rarity of the disease, CHMP deemed the adverse event profile acceptable, and designated Coagadex orphan medicine status. This designation gives drugmakers access to incentives such as fee reductions for scientific advice, and a key tool in the EU to encourage the development of medicines for patients with rare diseases.
With positive CHMP approvals generally mirrored by the European Commission, both Actelion and Bio Products will be hopeful of receiving full European approval in the near future.
Joel Levy
Related Content
Rethinking oncology trial endpoints with generalised pairwise comparisons
For decades, oncology trials have been anchored to a familiar set of endpoints. Overall survival …
Bristol Myers Squibb receives positive CHMP opinion for Opdivo treatment
Bristol Myers Squibb (BMS) has received a positive opinion from the Committee for Medicinal Products …
NICE issues positive final guidance for treatment of Duchenne Muscular Dystrophy from Santhera
Santhera Pharmaceuticals has received positive final guidance from the National Institute for Care and Excellence …
