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Expanded access plays an important role in the success of cell and gene therapy. Read our white paper which explores go-to-market models for success. https://lnkd.in/esNjw3if #ExpandedAccess #Gotomarket #MarketStrategy #PharmaMarketStrategy

Curious about expanded access in cell and gene therapies? Our whitepaper explores how this approach can bridge the gap between clinical trials and commercial availability. https://lnkd.in/eqtxypnE Learn how it's shaping the future of treatment access for patients with unmet medical needs. #ExpandedAccess #CellAndGeneTherapy #PatientCare

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> FDA widens Sarepta’s Duchenne gene therapy label to older boys in significant expansion of use: The FDA has broadened the label for Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy to patients ages 4 and older. The approval goes beyond the patient population in which the company reported data for its randomized clinical trials. It includes a full approval for Duchenne patients who are not dependent on a wheelchair, and an accelerated approval for those who are wheelchair-dependent. Marketed as Elevidys, the gene therapy was initially given accelerated approval last year for boys ages 4 and 5 who have Duchenne muscular dystrophy. It’s meant to slow progression of the aggressive muscle disease, and the expansion to older boys has long been awaited by families of patients who have few treatment options. It costs $3.2 million in the US and is not approved anywhere else in the world. There have been signs the FDA was going to expand the therapy’s label. Earlier this year, CBER Director Peter Marks made comments suggesting he was open to doing so. And when Elevidys was initially approved, Marks overruled FDA reviewers who had recommended rejecting it. Marks again overruled FDA staff in broadening the label of Sarepta’s gene therapy. Memos that were released Thursday with the agency’s decision showed that two top FDA officials, Lola Fashoyin-Aje and Nicole Verdun, recommended rejecting the gene therapy in favor of an additional confirmatory study but were overruled by CBER chief Peter Marks. It’s been debated whether the evidence has been strong enough to back the expansion. In October, the company reported that the Phase 3 EMBARK study failed to meet its primary endpoint. Intended as a confirmatory study, it showed that boys ages 4 to 7 who got Elevidys didn’t do better on a 17-item motor skill test than those who got placebo. However, the study met key secondary endpoints — measuring boys’ ability to stand and walk — that Sarepta cited when asking the FDA to approve the therapy for boys with Duchenne regardless of their wheelchair status or age. Continued approval for non-ambulatory Duchenne patients may depend on results from a confirmatory trial, which Sarepta said would be the Phase 3 ENVISION study with non-ambulatory and older ambulatory people with Duchenne. In its first six months on the market, Elevidys generated $200 million in revenue, reflecting strong demand for the therapy. The company’s stock $SRPT was up 35% in after-hours trading. It’s also been helped by the failure of a potential competitor: Last week, Pfizer announced that its Duchenne gene therapy did not succeed in a Phase 3 study. Pfizer’s therapy did not meet any of the secondary endpoints that Sarepta pointed to when asking for its label expansion. #lucidquest #genetherapy #celltherapy

#Cell & #GeneTherapy #ManufacturingServices Industry is projected to witness a CAGR of 19.9% during the period 2023-2032. This growth can be attributed to the increasing grants and investments for development of cell and gene therapies.

Gene&Cell Therapy >> FDA widens Sarepta’s Duchenne gene therapy label to older boys in significant expansion of use: The FDA has broadened the label for Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy to patients ages 4 and older. The approval goes beyond the patient population in which the company reported data for its randomized clinical trials. It includes a full approval for Duchenne patients who are not dependent on a wheelchair, and an accelerated approval for those who are wheelchair-dependent. Marketed as Elevidys, the gene therapy was initially given accelerated approval last year for boys ages 4 and 5 who have Duchenne muscular dystrophy. It’s meant to slow progression of the aggressive muscle disease, and the expansion to older boys has long been awaited by families of patients who have few treatment options. It costs $3.2 million in the US and is not approved anywhere else in the world. There have been signs the FDA was going to expand the therapy’s label. Earlier this year, CBER Director Peter Marks made comments suggesting he was open to doing so. And when Elevidys was initially approved, Marks overruled FDA reviewers who had recommended rejecting it. Marks again overruled FDA staff in broadening the label of Sarepta’s gene therapy. Memos that were released Thursday with the agency’s decision showed that two top FDA officials, Lola Fashoyin-Aje and Nicole Verdun, recommended rejecting the gene therapy in favor of an additional confirmatory study but were overruled by CBER chief Peter Marks. It’s been debated whether the evidence has been strong enough to back the expansion. In October, the company reported that the Phase 3 EMBARK study failed to meet its primary endpoint. Intended as a confirmatory study, it showed that boys ages 4 to 7 who got Elevidys didn’t do better on a 17-item motor skill test than those who got placebo. However, the study met key secondary endpoints — measuring boys’ ability to stand and walk — that Sarepta cited when asking the FDA to approve the therapy for boys with Duchenne regardless of their wheelchair status or age. Continued approval for non-ambulatory Duchenne patients may depend on results from a confirmatory trial, which Sarepta said would be the Phase 3 ENVISION study with non-ambulatory and older ambulatory people with Duchenne. In its first six months on the market, Elevidys generated $200 million in revenue, reflecting strong demand for the therapy. The company’s stock $SRPT was up 35% in after-hours trading. It’s also been helped by the failure of a potential competitor: Last week, Pfizer announced that its Duchenne gene therapy did not succeed in a Phase 3 study. Pfizer’s therapy did not meet any of the secondary endpoints that Sarepta pointed to when asking for its label expansion. #lucidquest #genetherapy #celltherapy

Read More: https://lnkd.in/ggyek-Wm #Cell & #GeneTherapyManufacturing Services Industry is projected to witness a CAGR of 19.9% from 2023 to 2032. This growth can be attributed to the increasing grants and investments for developing cell and gene therapies.

#Cell & #GeneTherapy Manufacturing Services Industry is projected to witness a CAGR of 19.9% during the period 2023-2032. This growth can be attributed to the increasing grants and investments for the development of cell and gene therapies.

#Cell & #GeneTherapyManufacturing Services Industry is projected to witness a CAGR of 19.9% during the period 2023-2032. This growth can be attributed to the increasing grants and investments for the development of cell and gene therapies. #GeneTherapy

Gene therapy is currently far from perfect, and one of the main concerns remains the safety of using these treatments. Patients who have undergone gene therapy often face autoimmune reactions, toxicity, and other adverse effects. I think the open FDA webinar, which you can register for, will provide a lot of new information and allow for discussion of key safety issues in gene therapy. I'm going!

We have made great strides at Muscular Dystrophy Association for the families we serve, with FDA-approved gene therapies for #SpinalMuscularAtrophy (#SMA) and #Duchenne #MuscularDystrophy (#DMD), however, challenges in development, manufacturing, and approval processes, particularly for #RareDiseases, must be addressed to make #GeneTherapies more accessible and sustainable. Read more in my article for Pharmacy Times, where I explore the latest advancements and challenges in #GeneTherapy for #neuromuscular diseases: https://lnkd.in/gzKiw_t6