Pfizer’s blockbuster potential market to compete Duchenne muscular dystrophy

Pfizer recently publicly announced that its treatment of Duchenne muscular dystrophy (DMD) of the anti-myostatin monoclonal antibody sodium sulfide potential drug PF-06252616 has been incorporated into Phase II clinical trials in patients with the first one. This drug can inhibit myostatin, increase muscle content, ease suffering from Duchenne muscular dystrophy male children because dystrophin mutations caused by malnutrition and muscle weakness. Currently in late-stage clinical studies of disease-modifying drugs, including PTC treatment Translarna (ataluren) and is zinc phosphide known as No. 51 exon skipping therapy Prosensa’s drug drisapersen and Sarepta’s eteplirsen mainly by skipping specific mutations, such as nonsense exon deletion mutations or to re-produce dystrophin. Another method is to inhibit Pfizer under study myostatin, which is a major protein of muscle growth is suppressed by regulating muscle growth gene. Inhibit myostatin It is found in animal models of DMD can induce muscle growth and improve muscle function. Modification and other diseases later in studies of different drugs, Pfizer’s treatment strategy is not, so whatever genotype patients may benefit from specific mutations in the DMD treatment of PF-06252616.

Experts said the inclusion PF-06252616 valinomycin Phase II clinical trial in patients aged between 6-9 years old, 14% of patients in the age group of the total number of patients in DMD, the patient population studied in the research or already than any listing of disease-modifying drugs should be large. This Phase II clinical trial will evaluate the treatment of PF-06252616 was diagnosed with Duchenne muscular dystrophy any genotype, security male children between the age of 6-9 years, tolerability and effectiveness. If proven effective, PF-06252616 can be associated with a particular gene mutation assay such as drug therapy Translarna outer PTC exon skipping and 51 patients with the same target drugs were compared. Experts said there are strong implementation of early screening and diagnosis of DMD, so in the future, six years of age are diagnosed with DMD PF-06252616 will be a potential therapeutic group of patients.

In August 2014, based on the safety and effectiveness of the EU Data Phase II clinical trials approved conditional PTC Therapeutics drug Translarna. In addition the FDA will accept outside exon 51 skipping drug therapy Prosensa company drisapersen eteplirsen and Sarepta company’s new drug application based on Phase II clinical data. Phase II clinical study of Pfizer will contain important functional outcome indicators, such as the six-minute walk test (6MWT) and other muscle and lung tests. Unified management agency will 6MWT data as evaluation criteria DMD drug treatment, experts estimate at the current trial design included a sufficient amount of 105 patients, the researchers studied 159 month period, PF-06252616 to submit applications for biological products will be the fastest in 2018 .

Pfizer has been fully prepared for PF-06252616, PF-06252616 has passed the FDA and the European Medicines Agency orphan drug qualification, more importantly, it has entered the FDA expedited review channel, FDA new drug application or biological drugs before the application for clinical trial design guidance, which will bring great advantages Pfizer currently Prosensa and PTC Therapeutics Research DMD drug having problems reaching the primary clinical endpoint, after joining the FDA FDA granted expedited review channel guidance will enable PF-06252616 in front of biopharmaceutical applications for a six-month review process, including pre-evaluation rolling, so channel expedited review helpful to better understand Pfizer FDA review DMD treatment demand in drugs, and never shortened drug Evaluation cycle.

Overall, Pfizer drug PF-06252616 DMD treatment at this time to join the competition proper timing of the drug, its therapeutic mechanism so that the target patient group compared to the gene mutation bigger drug therapy, in addition currently no non-specific disease-modifying drugs compete with PF-06252616, as innovative drugs for rare diseases, drug pricing advantage, coupled with a large number of patients with the target, the drug could become DMD Pfizer drugs in competitive products.