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Sarepta (SRPT) Gets FDA's Accelerated Nod for DMD Gene Therapy
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Sarepta (SRPT - Free Report) announced that the FDA has approved Elevidys (delandistrogene moxeparvovec or SRP-9001), an adeno-associated virus-based gene therapy to treat ambulatory pediatric patients aged between four and five years with Duchenne muscular dystrophy (“DMD”). Elevidys has been approved by the regulatory agency under the accelerated pathway.
Following the approval, Elevidys is the first one-shot gene therapy approved for treating DMD.A progressive and degenerative disorder, DMD leads to weakness and wasting away of the body’s muscles.
In a conference call with investors, Sarepta’s CEO Douglas Ingram stated that Elevidys will be priced at $3.2 million.
The FDA approval comes after several delays and questions over the effectiveness of Elevidys. Last month, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (“CTGTAC”) voted 8:6, narrowly recommending approval for Elevidys. This was likely due to the FDA’s concerns on Elevidys. Per the CTGTAC, the clinical studies conducted to date do not provide unambiguous evidence that the gene therapy will benefit DMD patients.
Based on the above factors, the FDA — in consultation with the company — decided to currently restrict the use of Elevidys in DMD patients aged between four and five years. The agency has stated that it will grant a non-age restricted expansion to Elevidysprovided the phase III EMBARK study achieves its objectives. The EMBARK study is the proposed confirmatory study that will seek full approval for Elevidys in DMD indication. Top-line results from the EMBARK study are expected in fourth-quarter 2023.
In the year so far, Sarepta’s shares have lost 4.4% compared to the industry’s 7.6% decline.
Image Source: Zacks Investment Research
Elevidys has been developed by Sarepta in collaboration with Roche (RHHBY - Free Report) . Both Sarepta and Roche entered into a licensing agreement in 2019 to jointly develop and commercialize Elevidys. Per the agreement, Roche has exclusive rights to launch and commercialize the gene therapy in ex-U.S. markets.
Sarepta’s commercial portfolio consists of three RNA-based PMO therapies targeting DMD — Exondys 51, Vyondys 53 and Amondys 45. These drugs can potentially address nearly a third of all patients with DMD in the United States.
Other than SRP-9001, Sarepta also develops SRP-5051 (vesleteplirsen), its next-generation exon-skipping pipeline candidate to treat DMD patients with skipping exon 51. This February, management also started an early-stage study (VOYAGENE) evaluating its other gene therapy candidate, SRP-9003, in patients with Limb-girdle muscular dystrophy (LGMD) type 2E/R4.
Sarepta currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the overall healthcare sector include ANI Pharmaceuticals (ANIP - Free Report) and Novartis (NVS - Free Report) . While ANI Pharmaceuticals sports a Zacks Rank #1 (Strong Buy), Novartis carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, the estimate for ANI Pharmaceuticals’ 2023 earnings per share (EPS) have increased from $2.42 to $3.31. During the same period, the earnings estimates for 2024 have risen from $3.76 to $4.32. Shares of ANI Pharmaceuticals are up 28.1% in the year-to-date period.
Earnings of ANI Pharmaceuticals beat estimates in each of the last four quarters, delivering an average earnings surprise of 68.64%.
In the past 60 days, the estimate for Novartis’ 2023 and 2024 EPS have increased from $6.57 to $6.74 and $7.08 to $7.28, respectively. Shares of Novartis are up 10.8% in the year-to-date period.
Earnings of Novartis beat estimates in each of the last four quarters, witnessing an average earnings surprise of 5.15%.
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Sarepta (SRPT) Gets FDA's Accelerated Nod for DMD Gene Therapy
Sarepta (SRPT - Free Report) announced that the FDA has approved Elevidys (delandistrogene moxeparvovec or SRP-9001), an adeno-associated virus-based gene therapy to treat ambulatory pediatric patients aged between four and five years with Duchenne muscular dystrophy (“DMD”). Elevidys has been approved by the regulatory agency under the accelerated pathway.
Following the approval, Elevidys is the first one-shot gene therapy approved for treating DMD.A progressive and degenerative disorder, DMD leads to weakness and wasting away of the body’s muscles.
In a conference call with investors, Sarepta’s CEO Douglas Ingram stated that Elevidys will be priced at $3.2 million.
The FDA approval comes after several delays and questions over the effectiveness of Elevidys. Last month, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (“CTGTAC”) voted 8:6, narrowly recommending approval for Elevidys. This was likely due to the FDA’s concerns on Elevidys. Per the CTGTAC, the clinical studies conducted to date do not provide unambiguous evidence that the gene therapy will benefit DMD patients.
Based on the above factors, the FDA — in consultation with the company — decided to currently restrict the use of Elevidys in DMD patients aged between four and five years. The agency has stated that it will grant a non-age restricted expansion to Elevidysprovided the phase III EMBARK study achieves its objectives. The EMBARK study is the proposed confirmatory study that will seek full approval for Elevidys in DMD indication. Top-line results from the EMBARK study are expected in fourth-quarter 2023.
In the year so far, Sarepta’s shares have lost 4.4% compared to the industry’s 7.6% decline.
Image Source: Zacks Investment Research
Elevidys has been developed by Sarepta in collaboration with Roche (RHHBY - Free Report) . Both Sarepta and Roche entered into a licensing agreement in 2019 to jointly develop and commercialize Elevidys. Per the agreement, Roche has exclusive rights to launch and commercialize the gene therapy in ex-U.S. markets.
Sarepta’s commercial portfolio consists of three RNA-based PMO therapies targeting DMD — Exondys 51, Vyondys 53 and Amondys 45. These drugs can potentially address nearly a third of all patients with DMD in the United States.
Other than SRP-9001, Sarepta also develops SRP-5051 (vesleteplirsen), its next-generation exon-skipping pipeline candidate to treat DMD patients with skipping exon 51. This February, management also started an early-stage study (VOYAGENE) evaluating its other gene therapy candidate, SRP-9003, in patients with Limb-girdle muscular dystrophy (LGMD) type 2E/R4.
Sarepta Therapeutics, Inc. Price
Sarepta Therapeutics, Inc. price | Sarepta Therapeutics, Inc. Quote
Zacks Rank & Stocks to Consider
Sarepta currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the overall healthcare sector include ANI Pharmaceuticals (ANIP - Free Report) and Novartis (NVS - Free Report) . While ANI Pharmaceuticals sports a Zacks Rank #1 (Strong Buy), Novartis carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, the estimate for ANI Pharmaceuticals’ 2023 earnings per share (EPS) have increased from $2.42 to $3.31. During the same period, the earnings estimates for 2024 have risen from $3.76 to $4.32. Shares of ANI Pharmaceuticals are up 28.1% in the year-to-date period.
Earnings of ANI Pharmaceuticals beat estimates in each of the last four quarters, delivering an average earnings surprise of 68.64%.
In the past 60 days, the estimate for Novartis’ 2023 and 2024 EPS have increased from $6.57 to $6.74 and $7.08 to $7.28, respectively. Shares of Novartis are up 10.8% in the year-to-date period.
Earnings of Novartis beat estimates in each of the last four quarters, witnessing an average earnings surprise of 5.15%.