Mesoblast Limited MESO announced that the FDA has approved Ryoncil (remestemcel-L) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in children aged two months and older, including adolescents and teenagers.

Following the FDA nod, Ryoncil became the first and only mesenchymal stromal cell therapy to be approved for any indication in the United States.

Ryoncil is also the first FDA-approved therapy for pediatric SR-aGvHD, a life-threatening condition with high mortality rates.

Shares of MESO were up 40.4% in after-hours trading on Dec. 18. The stock has skyrocketed 456.8% in the year so far against the industry’s decline of 10.5%.

Image Source: Zacks Investment Research

More Updates on MESO's Ryoncil

The FDA approval was based on data from the phase III study which evaluated Ryoncil for treating children with SR-aGvHD.

Data from the same showed that out of 89% of children who had high severity grade C or grade D disease, 70% achieved an overall response by day 28 following treatment with Ryoncil, a measure that predicts survival in aGVHD.

Treatment with Ryoncil was not discontinued at any point of the study due to any laboratory abnormality in the given patient population.

Currently, there are no approved therapies in the United States for treating patients under 12 years of age suffering from aGvHD, which should provide Mesoblast with a significant market opportunity.

Ryoncil is also being developed for additional inflammatory diseases including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease.

Competition in the Target Market

Incyte Corporation’s INCY lead drug, Jakafi, is approved for the treatment of SR-aGvHD in adult and pediatric patients aged 12 years and above in the United States. Jakafi is a first-in-class JAK1/JAK2 inhibitor.

Jakafi generated sales worth $2.02 billion in the first nine months of 2024. The drug has been witnessing a steady demand.

We note that Jakafi is marketed by Incyte in the United States and by Novartis NVS as Jakavi in ex-U.S. markets.

INCY receives royalty revenues from NVS for the commercialization of Jakavi in ex-U.S. markets.

Jakafi is also approved for chronic GVHD after failure of one or two lines of systemic therapy in adult and pediatric patients aged 12 years and older.

Incyte’s Jakafi was initially approved in the United States for treating adult patients with polycythemia vera (“PV”), who have had an inadequate response to or are intolerant to hydroxyurea. It is also approved for the treatment of adult patients with intermediate or high-risk myelofibrosis (“MF”), including primary MF, post-PV MF and post-essential thrombocythemia MF.

MESO's Zacks Rank & Key Pick

Mesoblast currently carries a Zacks Rank #3 (Hold).

A better-ranked stock from the biotech sector is Spero Therapeutics, Inc. SPRO, sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for Spero Therapeutics’ 2024 loss per share have narrowed from $1.59 to $1.29. During the same time, estimates for 2025 loss per share have narrowed from $1.54 to 79 cents. Year to date, shares of SPRO have lost 32.3%.

SPRO’s earnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average surprise of 94.42%.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report

Novartis AG (NVS) : Free Stock Analysis Report

Incyte Corporation (INCY) : Free Stock Analysis Report

Mesoblast Limited (MESO) : Free Stock Analysis Report

Spero Therapeutics, Inc. (SPRO) : Free Stock Analysis Report

To read this article on Zacks.com click here.

Zacks Investment Research