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Actions of Therapeutic Sarepta continue to decline in pre-commercial trade after the company announced Monday night that the US market Food and Drug Administration (FDA) dismissed the company's treatment against Duchene muscular dystrophy (DMD), golodirsen.
Sarepta said the The FDA has issued a full response letter regarding his request for a new drug that required accelerated approval of golodirsen injection in DMD patients with a confirmed mutation that could jump to exon 53. Sarepta anticipated that golodirsen would be the second treatment approved by the company. for the DMD.
Sarepta asked for approval based on the results of 4053-101, a phase I / II study that evaluated the safety, pharmacokinetics of tolerability, and the efficacy of golodirsen in 25 boys with confirmed deletions of the DMD gene likely to jump to exon 53. The test demonstrated statistically significant results in favor of golodirsen on all biological criteria, including a transcript of RNA jumped exon correctly using PCR (reverse transcription) , the amplitude of expression of dystrophin using Western blot and the intensity of dystrophin according to immunohistochemistry, explained Sarepta.
In the CRL, the FDA highlighted two concerns about treatment. The regulatory agency has expressed concern over the risk of intravenous infusion and port-related infections of kidney toxicity observed in preclinical models of golodirsen and observed after the administration of other oligonucleotides antisense, said Sarepta. In his announcement, Sarepta stated that renal toxicity with golodirsen has been observed in preclinical models at doses ten times greater than the dose used in clinical studies. However, the Cambridge, Massachusetts-based company said that renal toxicity was not observed in study 4053-101, on which the demand for golodirsen was based.
Doug Ingram, President and CEO of Sarepta, said the company was surprised to have received the LCR. Throughout the review with the FDA, Ingram stated that the regulatory agency had raised no issues suggesting "the non-amenability of golodirsen", including the issues raised in the letter . Ingram said the company would immediately request a meeting with the FDA to determine the next steps.
"We will work with the Division (FDA's Division of Neurological Products) to address the issues raised in the letter and, to the extent possible, find a fast track for golodirsen approval. We know that the patient community is waiting, "Ingram said in a statement.
Golodirsen is a morpholino-oligomeric phosphorodiamidate designed to treat patients with DMD who have genetic mutations that may skip the exon 53 of the dystrophin gene. This is comparable to the company's Exondys 51 drug, which was the first drug approved by the FDA for DMD. Exondys 51 skips the exon 51 and golodirsen is designed to ignore the exon 53 of the DMD gene. The exon skip is intended to allow the production of a truncated but functional dystrophin protein inside. The exon mutated 53 appears in approximately 8% of patients with DMD, which is the subgroup of DMD patients that golodirsen was to serve.
DMD is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in the function of muscle fibers. Progressive muscle weakness of the lower limbs extends to the arms, neck and other areas. Ultimately, increasing breathing difficulties due to respiratory muscle dysfunction require ventilation, and cardiac dysfunction can lead to heart failure. One of the most common fatal genetic disorders, DMD affects about one in every 3,500 babies born worldwide. The disease is universally fatal and death usually occurs before the age of 30.
Shares of Sarepta fell 3% on Monday to close at $ 120.31. This morning, stock prices fell by more than 17% in pre-market trading.
The Phase III ESSENCE study, which evaluates the efficacy and safety of golodirsen and casimersen, Sarepta exon-45 leaver, is underway. This test was to serve as a post-marketing study for golodirsen.
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