Apologies if already posted. Article from the Australian, taken from Yahoo Board.
The Australian.
Stem cell-focused biotech Mesoblast is seeking a meeting with the US Food and Drug Administration (FDA) in coming weeks in an effort to accelerate the approval of a drug aimed at saving children’s lives, while continuing to advance the same treatment to help fight COVID-19. The drug, remestemcel-L, has been designed to combat graft versus host disease (GvHD) in children but Mesoblast is hoping the drug can also win approval to treat people with serious coronavirus complications. Chief executive Silviu Itescu said the meeting next month with the FDA would be specifically about accelerating approval to treat GvHD in children under 12. He said remestemcel-L’s application on COVID-19 patients, will be discussed with the regulator at a later date. “We believe the immunomodulatory properties of remestemcel-L position this potential therapy at the forefront of treatment for severe and life-threatening inflammatory conditions, including COVID-19 acute respiratory distress syndrome (ARDS) and steroid-refractory acute graft versus host disease,” Dr Itescu said. “We are pursuing an accelerated approval pathway for remestemcel-L in the treatment of children with SR-aGVHD, and a parallel approval pathway for COVID-19 ARDS.” The meeting comes almost three months after the US Oncologic Drugs Advisory Committee voted nine to one in favour of the drug for use on GvHD - which is a crucial step towards full FDA approval. But last month, despite the advisory committee’s overwhelming vote in favour of the drug, the FDA asked Mesoblast to complete at least one more randomised, controlled study to ensure the treatment was safe and effective. Mesoblast is seeking to offer a compromise - fast track approval of remestemcel-L for children and the company will complete another trial for those over the age of 12. The reason being, it is difficult to enrol with a potentially fatal disease in a trial where you give some the treatment and others a placebo, effectively playing god. Dr Itescu said Mesoblast believes that remestemcel-L meets the criteria for accelerated approval as there are currently no approved treatments for this life-threatening condition in children under 12. “Children under 12 (with GvHD) currently have nothing approved and therefore we should be able to offer them something that the advisory panel has voted is effective,” Dr Itescu said. “We have offered the FDA that we will then do a trial in all of those people who have something approved, which is everyone over 12.” Separately, Mesoblast is enrolling patients in a phase three trial - the final step before regulatory approval and commercialisation - of remestemcel-L’s application in ventilator-dependent COVID-19 patients. The company is expecting the trial to be completed by February next year. Dr Itescu said the mortality rate among people suffering COVID-19 acute respiratory distress syndrome was still high - around 60 per cent - with few treatments available. “Whatever out there has fallen over,” Dr Itescu said. “The numbers are going up and it’s about whether our technology works or doesn’t work. We’ll see. Hopefully the phase three trial is positive.” Already, nine of 12 ventilator-dependent adult patients with COVID-19 ARDS who received two doses of remestemcel-L under emergency compassionate use at New York’s Mt Sinai Hospital were successfully discharged within 10 days. Dr Itescu said additionally two COVID-19 infected children with multisystem inflammatory syndrome (MIS-C) who received remestemcel-L for severe heart failure fully recovered heart function and were discharged within 30 hours of the second dose.
Key points 1. 60% morality rate for COVID ARDS. I think that correlates with Dr G's figures? 2. Agreed to do trial in patients over 12, but going for AA for those under 12 due to unmet need (no drugs avail.) and cannot play god with children. 3. "Whatever out there has fallen over" - few treatments available, meaning that MSB is now in the box seat for ARDS. 4. ARDS trial to be completed by Feb next year. Does that mean final dosage of last patients will be late-Jan + 30 days, or is "completing the trial" meaning having all results collated and submitted to FDA etc?
(20min delay)