Delving into the Latest Updates on Beremagene geperpavec with Synapse

Overview

Basic Info

Drug Type

Gene therapy

Synonyms

B-VEC, Bercolagene Telserpavec, beremagene geperpavec-svdt

+ [3]

Target

COL7A1

Mechanism

COL7A1 gene transference

Therapeutic Areas

Immune System DiseasesCongenital DisordersSkin and Musculoskeletal Diseases+ [1]

Active Indication

Epidermolysis Bullosa Dystrophica

Inactive Indication-

Originator Organization

Krystal Biotech, Inc.

Active Organization

Krystal Biotech, Inc.

Inactive Organization-

Drug Highest PhaseApproved

First Approval Date

US (19 May 2023),

Epidermolysis Bullosa Dystrophica

RegulationPRIME (EU), Paediatric investigation plan (EU), Orphan Drug (JP), Rare Pediatric Disease (US), Fast Track (US), Priority Review (US), Regenerative Medicine Advanced Therapy (US)

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Gene Sequence

Sequence Code 29659881

This is a 112-week (approximately two-year) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 2 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB). Participants will be dosed weekly with the topical B-VEC therapy. The primary endpoint will be to assess long term safety and tolerability of the topical gene therapy. The study is for those who participated in Phase 3 study, as well as, new participants who were unable to participate in the Phase 3 study, who meet all enrollment criteria.

Start Date25 May 2021

Sponsor / Collaborator

Krystal Biotech, Inc.

NCT04491604 / CompletedPhase 3

A Phase III Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, Previously "KB103") for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)

To determine whether administration of topical B-VEC improves wound healing as compared to placebo, and to evaluate durability, repeat dosing (Primary Endpoint) and further obtain safety and tolerability data.

Start Date17 Aug 2020

Sponsor / Collaborator

Krystal Biotech, Inc.

NCT03536143 / CompletedPhase 1/2

A Phase I/II Study of KB103, a Non-Integrating, Replication-Incompetent HSV Vector Expressing the Human Collagen VII Protein, for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)

This study was conducted to assess the safety and efficacy of topical Beremagene Geperpavec (KB103, HSV1-COL7) on DEB patients.

Start Date06 May 2018

Sponsor / Collaborator

Krystal Biotech, Inc.

100 Clinical Results associated with Beremagene geperpavec

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100 Translational Medicine associated with Beremagene geperpavec

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100 Patents (Medical) associated with Beremagene geperpavec

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14

Literatures (Medical) associated with Beremagene geperpavec

31 Dec 2024·Journal of Dermatological Treatment

Practical considerations relevant to treatment with the gene therapy beremagene geperpavec-svdt for dystrophic epidermolysis bullosa

Review

Author: Paller, Amy S. ; Marinkovich, M. Peter ; Lucky, Anne W. ; Ayala, Diego ; Guide, Shireen V. ; Gonzalez, Mercedes E. ; Bagci, Isin Sinem

BACKGROUND/PURPOSEDystrophic epidermolysis bullosa (DEB), a rare genetic skin disease caused by loss-of-function mutations in COL7A1, the gene encoding type VII collagen (COL7), is characterized by skin blistering, scarring, and extracutaneous manifestations that markedly reduce patient quality-of-life. Beremagene geperpavec-svdt ('B-VEC') is a gene therapy employing a non-integrating, replication-defective herpes simplex virus type 1 (HSV-1)-based vector encoding two copies of full-length human COL7A1 to restore COL7 protein after topical administration to DEB wounds. B-VEC was approved in the United States in 2023 as the first topical gene therapy and the first approved treatment for DEB. However, few providers have experience with use of this gene therapy.METHODSData was obtained through literature review and the experience of providers who participated in the B-VEC clinical study or initiated treatment after B-VEC approval.RESULTSThis review discusses the burden of disease, describes the clinical trial outcomes of B-VEC, and provides physician and patient/caregiver recommendations as a practical guide for the real-world use of B-VEC, which can be administered in-office or at the patient's home.CONCLUSIONSBy continuing to optimize the practical aspects of B-VEC administration, the focus will continue to shift to patient-centric considerations and improved patient outcomes.

01 May 2024·The Journal of Hand Surgery

Current Treatment Landscape for Dystrophic Epidermolysis Bullosa: From Surgical Management to Emerging Gene Therapies and Novel Skin Grafts

Review

Author: Fox, Paige M ; Perrault, David P ; Cholok, David ; Villavisanis, Dillan F ; Kiani, Sara N

Epidermolysis bullosa is a genetic skin disorder characterized by blister formation from mechanical trauma. Dystrophic epidermolysis bullosa (DEB) is caused by mutations in the COL7A1 gene presenting as generalized blisters from birth, which can result in extensive scarring, alopecia, esophageal stenosis, corneal erosions, and nail dystrophy. This disease also often leads to pseudosyndactyly of the digits from the closure of webspaces, progressing to a "mitten hand" deformity. Although traditional and current treatment for DEB is largely supportive with wound care and iterative surgical pseudosyndactyly release, emerging gene therapies and novel skin grafts may offer promising treatment. Studies published in the early 2020s have used HSV-1 vectors expressing missing COL7A1 genes to restore collagen function. One of these treatments, B-VEC, is an HSV-1-based topical gene therapy designed to restore collagen 7 by delivering the COL7A1 gene, leveraging a differentiated HSV-1 vector platform that evades the patient's immune system response. Other work has been performed to retrovirally modify autologous keratinocytes, but limitations of this process include increased labor in harvesting and engineering autologous cells. This article provides an overview of DEB treatment with an emphasis on emerging gene therapies and novel skin grafts, especially as they pertain to pseudosyndactyly treatment.

01 Mar 2024·JAMA Dermatology

Estimated Spending on Beremagene Geperpavec for Dystrophic Epidermolysis Bullosa

Article

Author: Mostaghimi, Arash ; Raymakers, Adam J N ; Kesselheim, Aaron S ; Feldman, William B

ImportanceNew gene therapies can offer substantial benefits to patients, particularly those with rare diseases who have few therapeutic options. In May 2023, the US Food and Drug Administration (FDA) approved the first topical gene therapy, beremagene geperpavec (B-VEC), for treating both autosomal recessive and autosomal dominant dystrophic epidermolysis bullosa (DEB). However, FDA approval was based on limited data in patients with autosomal dominant disease, even though they comprise approximately 50% of all DEB cases.ObjectiveTo estimate projected spending in the US on B-VEC therapy for treating autosomal recessive and autosomal dominant DEB.Design, Setting, and ParticipantsThis economic evaluation used data from the National Epidermolysis Bullosa Registry to estimate the current population of US patients with autosomal dominant and autosomal recessive DEB, with the aim of estimating US spending on B-VEC therapy from an all-payers perspective during 1- and 3-year periods after FDA approval. A base-case cost of $300 000 per patient per year was assumed based on a report from the manufacturer (Krystal Biotech).ExposureTreatment with B-VEC.Main Outcomes and MeasuresEstimated overall spending on B-VEC in the first year and over a 3-year period after FDA approval. Several prespecified sensitivity analyses with different assumptions about the eligible patient population and the cost of therapy were performed, and lifetime total costs of treatment per patient were estimated.ResultsThe estimated number of US patients with DEB who were eligible for treatment with B-VEC in the first year after FDA approval was 894. The estimated total expenditure for B-VEC therapy was $268 million (range, $179 million-$357 million). Over a 3-year period, estimated spending was $805 million (range, $537 million-$1.1 billion). Estimated lifetime total costs per patient were $15 million (range, $10 million-$20 million) per patient with autosomal recessive DEB and $17 million (range, $11 million-$22 million) for patients with autosomal dominant DEB.Conclusions and RelevanceResults of this economic evaluation suggest that the FDA’s broad indication for the use of B-VEC in treating both autosomal recessive and autosomal dominant DEB will have significant implications for payers.

86

News (Medical) associated with Beremagene geperpavec

12 Nov 2024

·www.globenewswire.com

Krystal Biotech to Present at Stifel 2024 Healthcare Conference

PITTSBURGH, Nov. 12, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced that the Company will participate in the Stifel 2024 Healthcare Conference on November 18, 2024, in New York. Krish S. Krishnan, Chairman and Chief Executive Officer, will take part in a fireside chat scheduled at 10:55 am ET and host investor meetings throughout the day. A webcast of the presentation will be available here beginning at 10:55 am ET on Monday, November 18, 2024 and will be posted on the Investors section of the Company’s website. About Krystal Biotech, Inc. Krystal Biotech, Inc. (NASDAQ: KRYS) is a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK® is the Company’s first commercial product, the first-ever redosable gene therapy, and the first medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and X (formerly Twitter). CONTACTInvestors and Media:Stéphane PaquetteKrystal Biotechspaquette@krystalbio.com

Drug ApprovalGene Therapy

06 Nov 2024

·www.globenewswire.com

Krystal Biotech to Present at Guggenheim Securities Healthcare Innovation Conference

PITTSBURGH, Nov. 06, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced that the Company will participate in the Guggenheim Securities Healthcare Innovation Conference on November 12, 2024, in Boston. Krish S. Krishnan, Chairman and Chief Executive Officer, will take part in a fireside chat scheduled at 1:30 pm ET and host investor meetings throughout the day. A webcast of the presentation will be available here beginning at 1:30 pm ET on Tuesday, November 12, 2024 and will be posted on the Investors section of the Company’s website. About Krystal Biotech, Inc. Krystal Biotech, Inc. (NASDAQ: KRYS) is a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK® is the Company’s first commercial product, the first-ever redosable gene therapy, and the first medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and X (formerly Twitter). CONTACTInvestors and Media: Stéphane PaquetteKrystal Biotechspaquette@krystalbio.com

Drug ApprovalGene Therapy

13 Sep 2024

·www.globenewswire.com

Krystal Biotech to Present at 2024 Cantor Global Healthcare Conference

PITTSBURGH, Sept. 13, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced that the Company will participate in the 2024 Cantor Global Healthcare Conference on September 18, 2024, in New York. Krish S. Krishnan, Chairman and Chief Executive Officer, will take part in a fireside chat scheduled at 1:20 pm ET and host investor meetings throughout the day. A webcast of the presentation will be available here beginning at 1:20 pm ET on Wednesday, September 18, 2024 and will be posted on the Investors section of the Company’s website. About Krystal Biotech, Inc.Krystal Biotech, Inc. (NASDAQ: KRYS) is a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK® is the Company’s first commercial product, the first-ever redosable gene therapy, and the first medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and X (formerly Twitter). CONTACTInvestors and Media:Stéphane PaquetteKrystal Biotechspaquette@krystalbio.com

Drug Approval

100 Deals associated with Beremagene geperpavec

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R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Epidermolysis Bullosa Dystrophica	US	Krystal Biotech, Inc.	19 May 2023

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Epidermolysis Bullosa Dystrophica	NDA/BLA	EU	Krystal Biotech, Inc.	01 Oct 2023

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04491604 (B-VEC-03, FDA) Manual	Phase 3	Epidermolysis Bullosa Dystrophica	-	VYJUVEK	ouckazsysk(tkfybforlx) = nmurxfshxi fpfpkimdpp (owudgigarg ) View more	Positive	19 May 2023
				Placebo	ouckazsysk(tkfybforlx) = xumxreygfj fpfpkimdpp (owudgigarg ) View more
NCT03536143 (KB103-001, FDA) Manual	Phase 1/2	Epidermolysis Bullosa Dystrophica	-	VYJUVEK	(qgxpauibgk) = Thirty-five treatment-emergent adverse events (TEAEs) were reported in 9 of the 12 subjects.Among the 35 TEAEs, the Applicant considers 19 as probable or possible drug-related AEs in five subjects. leekuvjbtf (tgqaqjrjsg )	Positive	19 May 2023
				Placebo
NCT04491604 (B-VEC-03 \| GEM-3, CTgov)	Phase 3	Epidermolysis Bullosa Dystrophica	31	Topical Beremagene Geperpavec (B-VEC)	uixqdbzupc(olcqcjywor) = aavfwjymtm wlmeubldry (zaiefuupsj, uprunplxjk - sztkyhwzry) View more	-	17 Feb 2023
				Placebo (Placebo)	uixqdbzupc(olcqcjywor) = pixndmgpur wlmeubldry (zaiefuupsj, aoplsxsqnn - aoltpyhqlg) View more
NCT03536143 (KB103-001 \| GEM-1, CTgov)	Phase 1/2	Epidermolysis Bullosa Dystrophica	12	Placebo gel (Placebo)	zbgvzvhcqo(jszrnayqhs) = sgyrjqiztr xqblkmcngb (lkmzcviykx, mubykmxlza - wynhgrmfks) View more	-	31 Jan 2023
				Topical beremagene geperpavec (Topical Beremagene Geperpavec)	vgjtqrvvfk(tksyjchobo) = ajhhrlktfk pvpphrhhph (jtrxmqfaau, yroayggzwl - fbtmmsnjax) View more
NCT04491604 (GEM-3, Corporate Publications) Manual	Phase 3	Epidermolysis Bullosa Dystrophica	62	Beremagene Geperpavec	znlothvzdg(hvqsattwzo) = qtnhqdbvjz rodlczlysy (tqwvebywkq ) View more	Positive	26 Mar 2022
				Placebo	znlothvzdg(hvqsattwzo) = jvgswpoawr rodlczlysy (tqwvebywkq ) View more