Penny stock moves forward with NDA for Duchenne treatment, seeks priority review – Endpoints News
After setbacks and delays, the Swiss biotech Santhera wants to get the treatment for Duchenne muscular dystrophy past the FDA.
Santhera, along with ReveraGen BioPharma, announced that it has completed the rolling submission for an NDA to US regulators and is seeking a priority review for vamorolone to treat Duchenne. Santhera states that the “core” of the NDA is the positive data from a phase IIb study showing the safety and effectiveness of the drug.
The results, published last year, examined dosage levels of 2 mg/kg per day and 6 mg/kg per day, and looked at how 121 patients’ time to stand up from lying flat was shortened after 24 weeks of treatment. In the high-dose arm, patients reduced their standing time from 6 seconds to 4.6 seconds, while the placebo group went from 5.4 to 5.5 seconds. This corresponded to a p-value of p=0.002. Santhera did not disclose the time difference for the lower dose, but reported a p-value of p=0.02.
So far, news of Santhera’s march against the NDA has piqued investors’ ears a bit, but the $SANN share price remains well down in the penny stock range as it faces a price drop of over 50% since last October.
“The completion of the vamorolone NDA submission is a major step toward our goal of bringing this investigational therapy to patients living with DMD, and represents an important milestone for Santhera,” Santhera CEO Dario Eklund said in a statement.
With Santhera applying for priority review, it expects the process to take 60 days if the review is granted and expects an approval date for some time in the middle of next year. Santhera also submitted its pitch to the EMA in September, seeking approval for vamorolone in the EU, Norway, Liechtenstein and Iceland.
Santhera has had a wild couple of years, as it was forced to halt a Phase III study in 2020 for its now-defunct idebenone program after it failed an interim analysis. The company had been preparing for a potential commercial launch across Europe and had to withdraw its marketing authorization application from the EMA. This led to most of the employees being made redundant in the subsequent restructuring.
After the Phase IIb gains for vamorolone in Duchenne, Santhera was extended a lifeline as it opened up some liquidity to enter the next year. However, Santhera hoped the FDA would consider the submission complete and accepted for review by late August or early September.