FDA delays review of Roche's new SMA drug Risdiplam

Roche's Gene Tekannounc announced on April 7 that the FDA had extended the approval date for Risdiplam's new drug application for spinal muscular dystrophy (SMA), with the PDUFA date being postponed by three months to August 24, 2020The delay was due to Genentech's recent submission of additional data from Risdiplam, including data from key research in SUNFISH Part 2, which worked closely with the FDANovember 2019, the FDA approved Risdiplam's priority review and decided to make a final decision by May 24 this yearIn February, after discussions with the FDA, Genentech again submitted additional data to help Risdiplam use it in a wider SMA populationThis includes 12-month efficacy and safety data from Part 2 of the SUNFISH study, the only placebo-controlled study ever conducted in patients aged 2 to 25 with type 2 or type 3 SMA, which showed that Risdiplam was significantly better at improving the patient's performance scoreThe FDA said it would need more time to review the volume of additional data submittedNo new SMA treatments have been approved since Spinraza was approved in 2016Both spinraza and Novartis' gene therapy Zolgensma are used in type 1 SMA patients, while Risdiplam will challenge the population of SMA patients who have not yet had a treatment option, as well as both drugs"The company is working closely with the FDA to support the review of Risdiplam," said DrLevi Garraway, chief medical officer and head of global product development atGenentechOur goal is to make this treatment available to infants, children and adults with SMA as soon as possible"
, Genentech has now submitted Risdiplam's listing documents to health regulators in six other countries around the world, and in December 2018 the European Medicines Agency (EMA) has granted Risdiplam priority drug eligibility, so the company is also expected to submit its application for market authorization for the drug to the EMA by mid-2020."SMA is a hereditary, neuromuscular disease that can lead to devastating muscle atrophy and disease-related complicationsIt is the most common genetic cause of infant death and one of the most common rare diseases, affecting about one in 11,000 babiesPatients with Type 1 SMA do not usually live to be over 2 years old, but mild type 2 and type 3 patients can get longer survival timesHowever, mild forms of the disease are still associated with mobility problems, respiratory infections and deathSMA is caused by a mutation in the genes of the surviving motor neuron 1 (SMN1), resulting in a lack of SMN proteinResearchers have found SMN proteins in the human body, and there is growing evidence that SMA is a multi-system disease, and that the loss of SMN proteins can affect many tissues and cells, potentially preventing human functionas part of a collaboration with the SMA Foundation and PTC Therapeutics, Genetek led the clinical development of Risdiplam, an oral liquid dosage form, a research-based SMA-surviving motor neuron 2 (SMN2) shearing modifier designed to consistently increase and maintain SMN protein levels throughout the central nervous system and tissues around the bodyIts potential to help the SMN2 gene produce more functional SMN proteins in the body is being evaluatedGenetek has designed an extensive clinical trial program for Risdiplam therapeutic SMA, with patients ranging from birth to age 60, including patients who have previously received other SMA targeted therapiesRisdiplam is currently being evaluated in four multicenter trials for SMA patients:SUNFISH (NCT02908685): a two-part, two-part, two-part, postbobono-controlled key study for type 2 or type 3 SMA patients between the ages of 2 and 25Part 1 identifies the dosage of the drug in Part 2 of the validation studyPart 2 used a total score of 32 (MFM-32) measurement of motion function during 12 months of treatment to assess the movement function slots of the head and limbs, sitting, standing and walking, upper limb strength and flexibility in SMA patientsMFM-32 is a validated scale for the evaluation of fine and overall motor function in patients with neurological disorders, including SMAThe study has reached its main destinationFIREFISH (NCT02913482): A two-part open label key clinical trial for type 1 SMA infantsPart 1 dose increment study of 21 infants was conducted primarily to assess the safety of Risdiplam in infants and to determine the dose of Part 2Part 2 of the 21 cases of Type 1 SMA infants treated for 24 months of single-arm study, followed by an open label extension period, registered in November 2018, the main goal is to assess the efficacy of Risdiplam, and through such as Bailey Infant Development Scale 3 (BSID-III) and Infant Exercise Scale assessment treatment after 12 months without support (defined as sitting without support 5 seconds)JEWELFISH (NCT03032172): Is an open label exploratory trial for SMA patients between 6 months and 60 years of age who have previously received SMA targeted treatment or olesoxime treatment and have completed their entry into the group RAINBOWFISH (NCT03779334): Is an open label, one-arm, multi-center study of the effectiveness, safety, pharmacokinetics and pharmacodynamics of infants genetically diagnosed with SMA symptoms from birth to six weeks of age (initial dose) and is currently being recruited reference source: 1, FDA sets back review for Roche's SMA drug by 3 months 2, FDA delays decision on Roche spinal sandatrophy drug