Re: "Only Sarepta." - De
December 13, 2019
Sarepta’s surprise FDA approval for rare disease drug excites investors
Shares of the biopharmaceutical jumped 35% after the Vyondys53 approval was announced Thursday evening
by Jaimy Lee
Marketwatch.com
The Food and Drug Administration’s out-of-the-blue approval of Sarepta Therapeutics’ second Duchenne muscular dystrophy (DMD) drug sent the company’s stock soaring on Friday.
Shares of Sarepta SRPT, +35.92% jumped 35% in morning trading after Thursday evening’s Vyondys 53 approval.
DMD is a rare disorder that mainly affects male children. About 8% of the children who have DMD have the gene mutation that Vyondys 53 treats.
The “Vyondys 53 approval removes a major overhang for the stock as concerns surrounding the future SRPT’s commercial products such as Exondys 51 being pulled from the market and cash flows required to support SRPT’s growing gene therapy platforms have now been lifted,” SVP Leerink’s Joseph Schwartz wrote on Dec. 12.
Sarepta already markets Exondys 51, which was the first FDA-approved DMD treatment in 2016. And until now, it has been the company’s only commercialized therapy, bringing in $99 million in sales in the third quarter, up from $78 million in the same period a year ago.
Until Aug. 19, when the regulator sent a complete response letter to the company citing a pair of risk concerns about the then experimental therapy, it was expected that Vyondys 53 would follow the same accelerated pathway as Exondys 51. The company’s stock tumbled 18% to $120.31 that day and has since failed to recover to its year-to-date high of $156.91 on July 15. The stock closed Thursday at $100.47. Company officials said they were surprised by the letter at that time.
“In the span of four months, we commenced and completed the formal dispute resolution process culminating in the grant of our appeal, resubmitted our [new drug application] and obtained an approval–a great benefit to DMD patients awaiting treatment,” Sarepta president and CEO Doug Ingram said in a news release.
A Sarepta spokeswoman said Exondys51 and Vyondys53 both have an annual list price of approximately $300,000. However, pharmacy benefit manager Prime Therapeutics told The New York Times in 2017 that the price can sometimes reach $750,000 per patient, in part because it is weight-based.
That price tag has raised questions among experts even though there are limited treatment options for DMD patients. The Institute for Clinical and Economic Review, a watchdog that conducts comparative clinical effectiveness research, said in August that most insurers have responded to the high price of Exondys 51 by narrowing coverage. “Three years after approval, the manufacturer has provided no high-quality evidence of benefit, many patients and families are surely losing out, and only the manufacturer truly benefits from this deplorable situation,” ICER chief medical officer David Rind said in a statement then.
Sarepta is also developing casimersen, another experimental DMD treatment that is expected to receive an FDA decision in the first half of next year. But not all analysts believe that the approval of Vyondys 53 bodes well for the FDA’s take on casimersen and other DMD medications in the industry pipeline.
“While the approval of [Vyondys 53] provides some alleviation to the P&L’s operational burn, we remain cautious of the next steps for development both for the PMO and gene therapy franchise,” wrote Oppenheimer analyst Hartaj Singh.
Investors are less cautious. Shares of Wave Life Sciences WVE, +2.67%, which is developing a DMD treatment, are up 6%. Audentes Therapeutics BOLD, +0.04%, which Astellas Pharma announced last week that it planned to buy for $3 billion, is also working on a DMD drug. Its stock was flat.
Sarepta’s stock has gained 25% year-to-date, while the S&P 500 SPX, +0.06% is up about 26%.
http://www.marketwatch.com/story/sareptas-surprise-fda-approval-for-rare-disease-drug-excites-investors-2019-12-13?siteid=yhoof2&yptr=yahoo
Gold is $1,581/oz today. When it hits $2,000, it will be up 26.5%. Let's see how long that takes. - De 3/11/2013 - ANSWER: 7 Years, 5 Months
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