Marathon Pharmaceuticals will hope to go the distance and not collapse at the end of its run as so many have before it after submitting its NDA for its Duchenne steroid drug deflazacort.
The drug, which is designed to treat patients with Duchenne muscular dystrophy (DMD)--the most common and most severe form of muscular dystrophy--will now be assessed by the FDA as to whether it will accept the filing. This process typically takes around 2 months.
The drug already has a slew of FDA tags, including: fast track status, orphan drug designation and dare pediatric disease designation for DMD.
The NDA filing is supported by both preclinical and clinical study programs, including two pivotal efficacy trials licensed by Marathon in more than 200 Duchenne patients 5 to 15 years of age.
These data, according to the Northbrook, ILL-based biotech, show that deflazacort improved muscle strength and other functional outcomes in patients with Duchenne regardless of genetic etiology--and in one of the studies ambulation status.
There is also an expanded access program, called “Access DMD”, ongoing in the U.S. that gives deflazacort to patients with Duchenne free of charge during the NDA review process.
“This NDA submission starts a process that we hope will result in broad access to this medication for all of those living with Duchenne who need it,” said Jeff Aronin, CEO Marathon Pharmaceuticals.
“We recognize the difficulty the Duchenne community has had in obtaining deflazacort and look forward to working closely with the FDA as they review our application.”
The difficulty Aronin speaks of has been compounded in recent months, with Sarepta ($SRPT) being knocked back by the FDA for its candidate eteplirsen (although an approval may still be on the cards as the FDA has asked for fresh data), and last month BioMarin ($BMRN) wrote off its $680 million Duchenne drug gamble for drisapersen, which had also been rejected by regulators.
And back in February, PTC Therapeutics' ($PTCT) also saw the FDA spurn its application for an approval of its DMD candidate Translarna (ataluren).
There are currently no therapies available to slow down a lethal condition that first cripples and then kills boys at an early age, leading to regulators--notably the FDA--coming under increasing pressure from families and carers of these boys to approve a new treatment.
Deflazacort works as a glucocorticoid with anti-inflammatory and immunosuppressant properties, and is a steroid with a long history outside of DMD and the U.S.
It’s currently off-patent across its global markets for its steroidal licenses, but Marathon will hope it can gain a U.S. approval in DMD--although the cost will likely rocket as a result. Many of these children are also treated with another steroid, prednisone, off-label, to help boost muscle strength and stave off respiratory and heart failure, as well as severe disability.
In one of the pivotal, randomized, double-blind, placebo controlled and active comparator studies, data showed that deflazacort met its primary endpoint of improved muscle strength versus placebo at 12 weeks.
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