The Committee for Medicinal Products for Human Use recommended approval of the 2mg/kg every-4-weeks dosing regimen in Fabry disease adult patients.
- The committee had issued a negative opinion on approving the new dosing regimen in October after it deemed the submitted data as insufficient to conclude on efficacy.
- Protalix said that it will be eligible to receive a regulatory milestone payment of $25 million from Chiesi Global Rare Diseases if the new dosing regimen is approved by the EMA.
Shares of Protalix BioTherapeutics, Inc. (PLX) closed 13% higher on Friday after it announced that a European Medicines Agency committee has recommended approval of a new dosing regimen for pegunigalsidase alfa, marketed as Elfabrio, in Fabry disease.
The Committee for Medicinal Products for Human Use recommended approval of the 2mg/kg every-4-weeks dosing regimen in Fabry disease adult patients. Currently approved dosing regimen requires patients to visit infusion centers every two weeks for treatment while the proposed new regimen would allow it to be stretched to every four weeks.
Turnaround
The positive opinion follows CHMP’s re-examination of the company’s application for the additional dosing regimen. The committee had issued a negative opinion on approving the new dosing regimen in October after it deemed the submitted data as insufficient to conclude on efficacy.
Protalix said that it will be eligible to receive a regulatory milestone payment of $25 million from Chiesi Global Rare Diseases if the new dosing regimen is approved by the EMA. The agency is expected to announce a decision by March 2026.
Fabry disease is a rare, inherited X-linked lysosomal storage disorder caused by a deficiency in the alpha-galactosidase A enzyme, leading to harmful accumulation of fats in cells. Symptoms include severe neuropathic pain in hands/feet, skin lesions, decreased sweating, and organ damage.
How Did Stocktwits Users React?
On Stocktwits, retail sentiment around PLX stock improved from ‘bullish’ to ‘extremely bullish’ territory over the past 24 hours, while message volume rose from ‘normal’ to ‘high’ levels.
A Stocktwits user appreciated that the company was persistent in getting a positive opinion. However, the stock should have rallied more on the news, they said.
“But the $25 million is great news and hopefully should lessen the risk of capital raise for their gout trials. Finally, one decent obstacle out of the way,” they wrote.
PLX stock has dropped about 2% over the past 12 months.
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