Cell Therapy News/Articles, page-5782

If this has already been posted.....apology in advance

Mesoblast scores US regulatory breakthrough
Tom Richardson and Carrie LaFrenz
Aug 14, 2020 – 7.31am
Save
Share
An advisory committee to the powerful US Food and Drug Administration (FDA) has voted nine to one in favour of a Mesoblast drug to treat transplant rejections in children.

The verdict was delivered by the Oncologic Drugs Advisory Committee (ODAC) about 7am AEST on Friday.

The committee voted the available clinical data supplied by Mesoblast supports the efficacy of its Ryoncil drug in pediatric patients with acute graft versus host disease (aGVHD), which can occur after a bone marrow transplant.

The final decision on whether to approve Mesoblast's Ryoncil drug for marketing in the US is now expected to be made by the FDA before September 30. If approved, the Australian-based regenerative medicine company will make Ryoncil immediately available in the US.

Shares in Mesoblast regained recent major losses, jumping 37.6 per cent to $4.65 immediately on Friday morning and ending the day up nearly 40 per cent.

The all-day meeting that included representations from Mesoblast's senior management team discussed both the adequacy of the product mechanism's delivery and whether a further clinical trial is needed to support the drug's efficacy.

MSBMesoblast Limited[/paste:font]$4.91 4.47%
1 year1 day

Sep 19Dec 19Mar 20Jun 201.002.003.004.005.00

Updated: Aug 17, 2020 – 4.03pm. Data is 20 mins delayed.View MSB related articles


Advertisement
The company also has an ongoing Phase 3 trial in progress to measure the effectiveness of a stem-cell therapy to treat ventilator-dependent COVID-19 patients. The first read out from an initial 90 patients involved in this trial is due by September.

Chief executive Silviu Itescu told investors it was too early to say if the product would be extended beyond children, but the group is working closely with the FDA.


Silviu Itescu has more than 20 per cent stake in Mesoblast, the company he founded and heads. AFR

"It was very important that the FDA received the advisory committee's confidence today," he said.

"It's also too early to state confidently what the label is going to say. But we are committed to working with community leaders and with the FDA, and seeking to have further data from an adult program to broaden the label beyond pediatrics to those adults with severe graft versus host disease."

Mesoblast chief medical officer Fred Grossman said there is clearly a significant un-met need in steroid-refractory aGVHD.

"We already have a plan to move forward with our adult protocol, and that should be under way in the very near future," he told analysts.

"We're very encouraged by today’s outcome and are committed to working
closely with the FDA as they complete their review of our submission regarding approval of Ryoncil for this life-threatening complication of an allogenic bone marrow transplant,” he added.


Advertisement
Dr Itescu said Mesoblast has built its leadership and sales team out in the US in anticipation of the FDA's decision as it gets ready to go to market. There is a team of 20 people to cover all regions, which will work with the various US payers, he said.

Industry sources have suggested a single course of the drug could cost around $US250,000 ($350,000) in the US. A course is thought to cost around $US180,000 in Japan, the only market with approval.

Dr Itescu added the vote in favour was a "very important step" for Mesoblast, which aims to launch multiple different stem cell products in the US to treat common inflammatory conditions.