just out on Sarepta puts an end to the speculation on their raisings, but they are still on very dodgy ground
2. SRP-9001By
Ben AdamsDrug: SRP-9001
Companies: Sarepta/Roche
Used for: Gene therapy for Duchenne muscular dystrophy
Est. 2028 sales: $2.2 billion
Duchenne muscular dystrophy (DMD) is a devastating disease that takes hold in childhood, slowly weakening the muscles including the heart. The disease, which only affects males, leaves young boys wheelchair-bound and statistically highly unlikely to see their fourth decade.
There remain few drug options for this disease with corticosteroids and physiotherapy becoming the standard therapy over the past few decades. Over the past six years, there have been newer, specific medicines for the condition, with three coming from
Sarepta Therapeutics alone, though these have come with questions over their efficacy and indeed whether some should have been approved at all.
The bigger overall issue is that Sarepta’s drugs target a small number of the total patient population. The ultimate goal in DMD treatment is to have a genuinely curative therapy that can help more patients, more widely; that is what
Sarepta, alongside drug partner Roche, hopes SRP-9001 can help kick-start.
Unlike Sarepta’s other approved drugs, SRP-9001 works as a gene therapy and is designed to deliver the microdystrophin-encoding gene into muscle tissue to prompt production of the microdystrophin protein. Patients with DMD have a mutation in the DMD gene and can’t make the protein on their own, leading to a progressive loss of muscle strength.
RELATEDFDA waves through a 3rd Sarepta DMD drug, once again based on questionable biomarker dataThough it could not be used by all patients, a gene therapy would be a vast improvement on the current drugs on the market. The promise of this therapy area remains high in and of itself as well as for future development, hence its high place on Evaluate’s list.
But despite being second,
with estimated 2028 sales of $2.2 billion, Evaluate cautions that U.S. commercial success for
SRP-9001 is by no means guaranteed. “Securing full reimbursement in the U.S. is likely to take time, should FDA approvals be won,” the analysts say in their 2023 report, adding this uncertainty “makes it hard to estimate how quickly sales” can be made.
This is especially true for such a new therapy area as “predicting demand is hard owing to lack of precedents and securing reimbursement for what will be a very expensive product is bound to present a problem,” the analysts add in their report. Gene therapy as a marketed treatment is still so new, how to handle it in terms of pricing and regulation will be a new path for everyone.
Questions also remain over this new way of treating DMD, notably on safety, an issue which has dogged gene therapies in the past, as well as its long-term efficacy.These therapies will also be expensive to make and to use, and that will also surely be reflected in their price, with the specter of drug pricing, value and affordability set to rear its head here.
Sarepta is still forging ahead, nabbing a speedy review with the FDA last year, with a Prescription Drug User Fee Act date of May 29, though this could be extended, or even be approved earlier.
And Sarepta is already making big moves to ensure it has the resources to bankroll the launch of SRP-9001, should the FDA give its OK later this year.
The biopharma’s CEO Doug Ingram said in a November interview with Fierce Biotech that the company raised more than a billion dollars during the third quarter “to ensure we have the resources necessary to fully prepare for and successfully launch SRP-9001.”The company is also changing its commercial, medical affairs, access and reimbursement and patient services teams to prepare for “what could be the most consequential gene therapy launch in history,” Ingram added in the interview.
So it just goes to show the cost involved post approval, they have an arsenal of over $1bil to carry their drug forwards to succesful launch.
Not sure where ANP sits in all of this , we cant even raise the equvelant of our current MC never mind a billion Bucks to market it LOL
If this new CEO is not well connected Heaven Help US.
https://www.fiercepharma.com/marketing/top-10-most-anticipated-drug-launches-2023
