Alexion’s European Approval And Other News: The Good, Bad And Ugly Of Biopharma – Seeking Alpha

Alexion Pharma Receives European Approval for Ultomiris

Alexion Pharmaceuticals (ALXN) announced that its lead drug candidate Ultomiris has been approved by the European Commission. Ultomiris may now be used for treating adults and children with atypical hemolytic uremic syndrome in Europe. The drug candidate is the first and only long-acting C5 complement inhibitor that needs to be administered every eight weeks.

The European Commission based its data on two global, single arm studies of the drug candidate. One of these studies was conducted in adults while the other was conducted in children. Both the trials are still operational. The interim analysis involved 56 out of 58 complement inhibitor treatment-nave adults and 18 out of 21 complement inhibitor treatment-nave children. For the initial 26-week treatment periods, 54 percent of adults and 77.8 percent (interim data) of children showed Complete TMA Response.

In the 52-week follow-up period, 4 more adult patients and 3 pediatric patients demonstrated a Complete TMA Response that was validated after the 26-week Initial Evaluation Period. This additional data takes the overall Complete TMA Response of 61 percent in adults and 94 percent in children as shown in interim data.

The drug is expected to create a niche for itself in the market with its convenient dosage regimen of eight weeks. John Orloff, M.D., of Alexion said, Treatment with ULTOMIRIS offers convenient eight-week dosing, which we believe is preferred by patients because it provides them with greater flexibility and improved quality of life, while also reducing the burden on healthcare systems that currently face significant strain in many countries. Atypical HUS is an ultra-rare disease which impacts vital organs such as kidneys by damaging walls of blood vessels.

The company has established a Risk Management Plan for ULTOMIRIS for minimizing the risk of side effects for patients. The most frequently observed adverse reactions related to the drug were nausea, upper respiratory tract infection and headache, among others. ULTOMIRIS is known to cause meningococcal infections, however, aHUS studies did not report any such incident.

ULTOMIRIS was approved by the FDA in 2019 for treating aHUS in adult and pediatric patients of one month of age and older. The drug is also approved in the United States and Japan for treating adult patients suffering from paroxysmal nocturnal hemoglobinuria. In Europe, the drug is authorized for treating patients with PNH with hemolysis with clinical symptoms indicative of high disease activity. The companys application for marketing authorization as a treatment for aHUS is currently under review in Japan.

ULTOMIRIS works by controlling the C5 protein in the terminal complement cascade. The treatment is designed to be administered intravenously every eight weeks or every four weeks for pediatric patients less than 20 kg, following a loading dose.

Alexion is a biopharmaceutical firm and focuses on complement biology and inhibition. The company has two approved complement inhibitors and two enzyme replacement therapies in its portfolio. It also has robust development pipeline with a number of prospective therapies in advanced trial stages. The company seeks to work on developing novel molecules and their impact on core therapeutic areas such neurology and nephrology.

Intercept Pharmaceuticals Inc. (ICPT) reported that it has received a Complete Response Letter from the FDA with regard to its Fibrosis drug. The letter indicates that agency has found the predicted benefits of the drug candidate to be uncertain. The FDA has recommended the company to submit post-interim analysis efficacy and safety data. The FDA stated that it felt that the modest fibrosis effect was not clearly predictive of clinical efficacy,

Intercept had earlier indicated that it expected the review may take longer than the previously announced PDUFA goal date as the agency had postponed a tentatively planned advisory committee meeting. Mark Pruzanski, M.D.,CEO of Intercept said, At no point during the review did the FDA communicate that OCA was not approvable on an accelerated basis, and we strongly believe that the totality of data submitted to date both meet the requirements of the Agencys own guidance and clearly support the positive benefit-risk profile of OCA. The FDA has recommended the company to continue the long-term outcome phase of its trial.

The NDA submission for obeticholic acid is the first for NASH. The application was backed by the data collated from 35 clinical trials. OCA is the only investigational NASH drug to demonstrate replicable ability to overturn or stabilize liver fibrosis in patients with advanced fibrosis due to NASH. The FDA has asked Intercept to submit additional data from its ongoing REGENERATE study to support its potential accelerated approval pathway.

There is no approved therapy for treating NASH fibrosis yet. OCA has been given Breakthrough Therapy designation by the FDA. The OCA is already approved by the FDA for treating primary biliary cholangitis.

Ascendis Pharma A/S (ASND) announced that it has submitted a Biologics License Application to the FDA seeking label expansion for TransCon. The company is looking to add the treatment of pediatric growth hormone deficiency to its label. Currently, there is no approved long-acting growth hormone treatment available in the United States or Europe.

TransCon hGH is a long-acting once-weekly prodrug of somatropin and aims to work on the same mode of action as daily daily hGH therapies. The drug candidate provides the therapeutic benefits by releasing the same growth hormone molecule, somatropin. Jan Mikkelsen, CEO of Ascendis said, We believe the TransCon technology has the potential to address major unmet medical needs and we look forward to continuing the late stage clinical development of our other endocrinology rare disease product candidates, TransCon PTH and TransCon CNP, as well as filing an Investigational New Drug application for our first oncology product candidate later this year.

The company plans to file a Marketing Authorization Application for the drug candidate in Europe during the third quarter. The drug candidate has orphan designation for GHD in both the United States and Europe. Ascendis plans to begin a new Phase 3 trial with TransCon hGH in pediatric GHD in Japan. The trial will likely be initiated in the fourth quarter. Another Phase 3 trial is currently underway in Greater China. Ascendis is collaborating with VISEN Pharmaceuticals for this purpose.

TransCon denotes Transient Conjugation and is a proprietary technology platform for producing more efficient therapies. The three main components of a TransCon molecule are an unmodified parent drug, an inert carrier to protect it and a liker to temporarily bind the two.

Ascendis Pharma has strong development pipeline with three independent endocrinology rare disease product candidates. The company is now looking to expand its focus on oncological segment.

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Alexion's European Approval And Other News: The Good, Bad And Ugly Of Biopharma - Seeking Alpha