Rare Pediatric Disease Priority Voucher

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Rare Pediatric Disease (RPD) Designation and Voucher ...

(11 days ago) Rare Pediatric Disease (RPD) Designation and Voucher Programs Under Section 529 to the Federal Food, Drug, and Cosmetic Act (FD&C Act), FDA will award priority review vouchers to sponsors of rare...

https://www.fda.gov/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs

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Rare Pediatric Disease Priority Review Vouchers | FDA

(3 days ago) Under section 529, FDA will award priority review vouchers to sponsors of certain rare pediatric disease product applications that meet the criteria specified in that section. FDA’s guidance...

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/rare-pediatric-disease-priority-review-vouchers

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Issuance of Priority Review Voucher; Rare Pediatric ...

(5 months ago) The Food and Drug Administration (FDA) is announcing the issuance of a priority review voucher to the sponsor of a rare pediatric disease product application.

https://www.federalregister.gov/documents/2020/09/15/2020-20320/issuance-of-priority-review-voucher-rare-pediatric-disease-product

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Rare Pediatric Disease Priority Review Vouchers

(3 days ago) Note that there are limitations on when rare pediatric disease priority review vouchers can be awarded: FDA may not award a voucher if the application was submitted to FDA prior to October 7, 2012 ...

https://www.fda.gov/media/90014/download

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FDA updates rare pediatric disease draft guidance

(1 months ago) “One program in place intended to advance the development of drugs and biologics for certain serious and life-threatening rare pediatric diseases is the Rare Pediatric Disease Priority Review...

https://www.fda.gov/news-events/fda-brief/fda-brief-fda-updates-draft-guidance-rare-pediatric-disease-priority-review-voucher-program

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Home - Priority Review Vouchers

(2 days ago) The Food and Drug Administration (FDA) awards a voucher following approval of a treatment for a neglected disease, rare pediatric disease, or medical countermeasure. Two drugs receive priority review for each voucher: the drug winning a voucher for a neglected or rare pediatric disease, and the drug using a voucher for another indication.

https://sites.fuqua.duke.edu/priorityreviewvoucher/

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Priority Review Vouchers General Information and Redemption

(20 days ago) Rare pediatric disease means it is • A serious or life threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years and

https://www.fda.gov/media/135235/download

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Should the FDA Renew its Rare Pediatric Disease Priority ...

(6 days ago) The goal of the FDA’s Rare Pediatric Disease Priority Review Voucher Program (PRV) is to stimulate treatment development and expedite patient access to these treatments.

https://www.pharmalawgrp.com/blog/3/should-the-fda-renew-its-rare-pediatric-disease-priority-review-voucher-program/

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Rhythm Pharmaceuticals Announces Sale of Priority Review ...

(3 days ago) About the Rare Pediatric Disease Priority Review Voucher Program The program is intended to encourage development of new drug and biological products for prevention and treatment of certain rare...

https://www.globenewswire.com/news-release/2021/01/05/2153459/0/en/Rhythm-Pharmaceuticals-Announces-Sale-of-Priority-Review-Voucher-for-100M.html

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Regulatory Explainer: Everything You Need to Know About ...

(3 days ago) The rare pediatric voucher was created in 2012 under the Food and Drug Administration Safety and Innovation Act (FDASIA), and specifically targets the need for additional therapies for rare pediatric subsets of other diseases.

https://www.raps.org/regulatory-focus/news-articles/2017/12/regulatory-explainer-everything-you-need-to-know-about-fdas-priority-review-vouchers

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US FDA Grants Rare Pediatric Disease Designation (RPDD) to ...

(3 days ago) With RPDD granted, Kazia may now be eligible to receive a 'rare pediatric disease priority review voucher' (PRV) if paxalisib is approved for DIPG A PRV grants the holder an expedited six-month ...

https://finance.yahoo.com/news/us-fda-grants-rare-pediatric-003000629.html

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The FDA Is Ending Its Rare Pediatric Disease Priority ...

(2 days ago) The rare pediatric disease priority review voucher program is one incentive that’s helped to spur research and development in this area with more than 20 vouchers granted to date, but it is set to expire on September 30, 2020.

https://www.regprofessional.com/the-fda-is-ending-its-rare-pediatric-disease-priority-review-vouchers-and-time-is-running-out-to-get-one/

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Notice of Approval of Product Under Voucher: Rare ...

(15 days ago) Under section 529 of the FD&C Act (21 U.S.C. 360 ff), which was added by FDASIA, FDA will report the issuance of rare pediatric disease priority review vouchers and the approval of products for which a voucher was redeemed. FDA has determined that BEOVU (brolucizumab-dbll), approved October 7, 2019, meets the redemption criteria.

https://www.federalregister.gov/documents/2019/12/16/2019-27054/notice-of-approval-of-product-under-voucher-rare-pediatric-disease-priority-review-voucher

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Fee for Using a Rare Pediatric Disease Priority Review ...

(5 days ago) The recipient of a rare pediatric disease priority review voucher may either use the voucher for a future human drug application submitted to FDA under section 505 (b) (1) of the FD&C Act (21 U.S.C. 355 (b) (1)) or section 351 (a) of the Public Health Service Act (42 U.S.C. 262 (a)), or transfer (including by sale) the voucher to another party.

https://www.federalregister.gov/documents/2019/09/30/2019-21197/fee-for-using-a-rare-pediatric-disease-priority-review-voucher-in-fiscal-year-2020

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Issuance of Priority Review Voucher; Rare Pediatric ...

(11 days ago) Under section 529 of the FD&C Act (21 U.S.C. 360 ff), which was added by FDASIA, FDA will award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria.

https://www.federalregister.gov/documents/2018/12/13/2018-27043/issuance-of-priority-review-voucher-rare-pediatric-disease-product

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Rhythm Pumps $100M From Voucher Sale Into Genetic Obesity ...

(2 days ago) In a $100 million deal, Rhythm Pharmaceuticals is selling its rare pediatric disease priority review voucher (PRV), granted by the U.S. Food and Drug Administration (FDA) with the approval of Imcivree (setmelanotide) to treat three rare genetic obesity disorders.

https://geneticobesitynews.com/2021/01/11/rhythms-pumps-100m-priority-review-voucher-sale-into-imcivre/

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Congress Approves a Four Year Extension of the Rare ...

(6 days ago) The rare disease community realized an important policy victory as Congress approved a four year extension of the Rare Pediatric Disease Priority Review Voucher (PRV) Program, capping off a year of community driven advocacy for the Creating Hope Reauthorization Act.

https://everylifefoundation.org/congress-approves-a-four-year-extension-of-the-rare-pediatric-disease-priority-review-voucher-prv-program/

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GW Pharma announces sale of priority review voucher for ...

(1 months ago) GW Pharmaceuticals Plc GWPH, +1.19% said Monday it is selling its Rare Pediatric Disease Priority Review Voucher for $105 million. The company, which was the first to receive U.S. Food and Drug ...

https://www.marketwatch.com/story/gw-pharma-announces-sale-of-priority-review-voucher-for-105-million-2019-03-18-7914623

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Impact Of The Priority Review Voucher Program On Drug ...

(3 months ago) Only an estimated 5 percent of rare pediatric diseases have a treatment, although collectively they affect more than ten million children in the US. To stimulate drug development for rare pediatric diseases, Congress expanded the priority review voucher (PRV) program in 2012. A pediatric PRV, which …

https://pubmed.ncbi.nlm.nih.gov/30715972/

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U.S. GAO - Drug Development: FDA's Priority Review Voucher ...

(3 days ago) Drugs that treat certain tropical diseases, rare pediatric diseases and illnesses related to public health emergencies are essential—but they may not be the most profitable to make.When drug developers make these kinds of drugs, FDA can award them “priority review vouchers.” Drug developers can redeem a voucher to speed up FDA review of a future—potentially more profitable—drug.

https://www.gao.gov/products/GAO-20-251

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Creating Hope Reauthorization Act (2020 - H.R. 4439)

(4 days ago) Sep 29, 2020. H.R. 4439 (116th). To amend the Federal Food, Drug, and Cosmetic Act to extend the authority of the Secretary of Health and Human Services to issue priority review vouchers to encourage treatments for rare pediatric diseases. In GovTrack.us, a database of bills in the U.S. Congress.

https://www.govtrack.us/congress/bills/116/hr4439

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Forge Biologics Receives FDA Fast Track, Orphan Drug, and ...

(7 days ago) On December 27, 2020, the Rare Pediatric Disease Priority Review Voucher Program was extended by Congress after it was scheduled to sunset in 2020. Under the newly extended RPD program, if FBX-101 ...

https://www.prnewswire.com/news-releases/forge-biologics-receives-fda-fast-track-orphan-drug-and-rare-pediatric-disease-designations-for-fbx-101-gene-therapy-for-patients-with-krabbe-disease-301228668.html

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What happened to the value of priority review vouchers ...

(5 days ago) The Creating Hope Act in 2012 substantially expanded the PRV program to include rare pediatric diseases. Last week Sarepta Therapeutics announced the sale of its priority review voucher, awarded by the FDA following the approval of Exondys 51 (eteplirsen) for Duchenne muscular dystrophy in September 2016.

https://www.locustwalk.com/what-happened-to-the-value-of-priority-review-vouchers-prv/

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Priority review - Wikipedia

(2 days ago) Priority review vouchers are currently earned by pharmaceutical companies for the development and approval of drugs treating neglected tropical diseases, rare pediatric diseases, and "medical countermeasures" for terrorism. The voucher can be used for future drugs that could have wider indications for use, but the company is required to pay a ...

https://en.m.wikipedia.org/wiki/Priority_review_voucher

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Taysha Gene Therapies Receives Rare Pediatric Disease and ...

(13 days ago) The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations.

https://www.businesswire.com/news/home/20201203005269/en/Taysha-Gene-Therapies-Receives-Rare-Pediatric-Disease-and-Orphan-Drug-Designations-for-TSHA-103-for-the-Treatment-of-Epilepsy-Caused-by-SLC6A1-Haploinsufficiency

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§360ff. Priority review to encourage treatments for rare ...

(20 days ago) The term "priority review voucher" means a voucher issued by the Secretary to the sponsor of a rare pediatric disease product application that entitles the holder of such voucher to priority review of a single human drug application submitted under section 355(b)(1) of this title or section 351(a) of the Public Health Service Act [42 U.S.C. 262 ...

https://uscode.house.gov/view.xhtml?req=(title:21%20section:360ff%20edition:prelim)

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Rare Diseases | Regulation | Pink Sheet

(4 days ago) BioMarin Pharmaceutical Inc. 's latest priority review voucher sale is further evidence that the price for the coupons, which entitle the holder to a speedier US FDA review, is leveling off. On Nov. 27, BioMarin announced plans to sell a rare pediatric disease priority review voucher to an undisclosed purchaser for a lump sum payment of $125m.

https://pharmaintelligence.informa.com/resources/product-content/voucher-sales-prices

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Bill Proposes Continuation of FDA Rare Pediatric Disease ...

(4 days ago) To help stimulate new development of rare pediatric disease (RPD) drugs, Congress created the RPD Priority Review Voucher Program in 2012, which offered additional incentives for obtaining FDA approval of these products.

https://lifesciences.mofo.com/topics/Bill-Proposes-Continuation-of-FDA-Rare-Pediatric-Disease-Priority-Review-Vouchers.html

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Rhythm Pharmaceuticals Announces Sale of Priority Review ...

(6 days ago) Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that it has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $100M.

https://www.biospace.com/article/releases/rhythm-pharmaceuticals-announces-sale-of-priority-review-voucher-for-100m/

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Bill Proposes Continuation Of FDA Rare Pediatric Disease ...

(4 days ago) The House of Representatives recently passed a proposed bill that encourages treatments for rare pediatric diseases by prolonging the authority of Health and Human Services to issue priority review vouchers. H.R.4439, or the "Creating Hope Reauthorization Act," aims to amend Section 529(b)(5) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff(b)(5)) to extend the program cutoff ...

https://www.mondaq.com/unitedstates/food-and-drugs-law/995940/bill-proposes-continuation-of-fda-rare-pediatric-disease-priority-review-vouchers

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Priority Review Vouchers for rare pediatric diseases ...

(10 days ago) RARE PEDIATRIC DISEASE DRUG NAME COMPANY VOUCHER AWARD YEAR AND SALE AMOUNT BUYER; 1. Morquio A syndrome: Vimizim : BioMarin: 2014, $67.5M: Regeneron Pharmaceuticals Inc. and Sanofi SA, redeemed for Praluent 2.

https://www.kidsvcancer.org/priority-review-vouchers/

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Short Sunsets for Priority Review Voucher Programs May ...

(2 days ago) The voucher program works by granting a priority review voucher to a drug developer who develops and gains approval for a therapy that treats a neglected tropical disease, a rare pediatric disease, or that is a medical countermeasure.

https://www.bio.org/blogs/short-sunsets-priority-review-voucher-programs-may-mitigate-incentive

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Editas' SCD Candidate Gets FDA's Rare Pediatric Disease ...

(6 days ago) Additionally, the RPD status conferred on the candidate will provide Editas with an opportunity to obtain a Pediatric Disease Priority Review Voucher from the FDA after the regulatory body ...

https://www.nasdaq.com/articles/editas-scd-candidate-gets-fdas-rare-pediatric-disease-tag-2020-08-25

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Mullin Discusses Importance of Investing in Rare Pediatric ...

(15 days ago) Without Congressional action, the Rare Pediatric Disease Priority Review Voucher Program is set to expire at the end of the fiscal year. The elimination of the Rare Pediatric Disease PRV program could have devastating impacts on rare disease drug development moving forward.

https://mullin.house.gov/news/documentsingle.aspx?DocumentID=4251

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Pediatric Rare Disease Voucher Program Extended

(6 days ago) Congress recently provided a short-term extension until December 11 of the Rare Pediatric Disease Priority Review Voucher program. The program was set to expire on September 30, but was given a reprieve in the Continuing Appropriations Act, 2021 and Other Extensions Act (H.R. 8337) that kept the government funded.

https://bioutah.org/pediatric-rare-disease-voucher-program-extended/

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Retrotope Granted Rare Pediatric Disease Designation from ...

(1 days ago) Under the FDA’s rare pediatric disease priority review voucher program, a sponsor that receives an approval for a drug that has been granted rare pediatric disease designation may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product or sold to other companies.

https://apnews.com/press-release/globenewswire-mobile/business-products-and-services-mental-health-product-approvals-product-testing-362e7ed89bae1b2846222e7ec2d37199

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Ultragenyx Sells Priority Review Voucher for $130 million ...

(5 days ago) About the Rare Pediatric Disease Priority Review Voucher Program This U.S. FDA program is intended to encourage development of new drug and biological products for prevention and treatment of ...

https://www.globenewswire.com/news-release/2017/12/18/1263363/0/en/Ultragenyx-Sells-Priority-Review-Voucher-for-130-million.html

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FDA Grants Rare Pediatric Disease, Orphan Drug Designation ...

(2 days ago) Taysha’s TSHA-105 fell under the agency’s Rare Pediatric Disease Priority Review Voucher Program, which aims to “address the challenges that drug companies face when developing treatments for these unique patient populations,” according to the company’s statement about the designations.

https://www.biospace.com/article/fda-grants-rare-pediatric-disease-orphan-drug-designation-for-taysha-s-rare-epilepsy-candidate/

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Sarepta Therapeutics Agrees to Sale of Priority Review ...

(3 days ago) About the Rare Pediatric Disease Priority Review Voucher Program The program is intended to encourage development of new drug and biological products for prevention and treatment of certain rare pediatric diseases.

https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-agrees-sale-priority-review-voucher-125m

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Bill Proposes Continuation Of FDA Rare Pediatric Disease ...

(5 days ago) To help stimulate new development of rare pediatric disease (RPD) drugs, Congress created the RPD Priority Review Voucher Program in 2012, which offered additional incentives for obtaining FDA ...

https://www.jdsupra.com/legalnews/bill-proposes-continuation-of-fda-rare-81623/

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Tropical Or Rare Pediatric Disease Priority Review ...

(6 days ago) Tropical or Rare Pediatric Disease Priority Review Vouchers: Update and Use of the 505(b)(2) Pathway. While the United States market for drugs and biologics targeting tropical or rare pediatric diseases is very small, developing novel therapies for patients in these underrepresented groups can be advantageous for both patients and Sponsors.

https://camargopharma.com/resources/blog/tropical-rare-pediatric-disease-priority-review-vouchers-update-use-505b2-pathway/

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GW Pharmaceuticals plc Announces the Sale of Priority ...

(10 days ago) About the Rare Pediatric Disease Priority Review Voucher Program The program is intended to encourage development of new drug and biological products for prevention and treatment of certain rare ...

https://www.globenewswire.com/news-release/2019/03/18/1756217/0/en/GW-Pharmaceuticals-plc-Announces-the-Sale-of-Priority-Review-Voucher-for-105M.html

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Spark Therapeutics Receives Rare Pediatric Disease ...

(6 days ago) Under FDA's Rare Pediatric Disease Priority Review Voucher program, companies who receive approval for a new drug application or Biologics License Application (BLA) for a rare pediatric disease ...

https://www.globenewswire.com/news-release/2017/07/20/1054633/0/en/Spark-Therapeutics-Receives-Rare-Pediatric-Disease-Designation-for-Investigational-LUXTURNA-voretigene-neparvovec-from-FDA.html

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Chinook Receives Rare Pediatric Disease Designation from U ...

(2 days ago) Through the FDA’s Rare Pediatric Disease Designation and Voucher Programs, the FDA may grant a priority review voucher at the time of product approval for a “rare pediatric disease.” The ...

https://www.globenewswire.com/news-release/2021/02/02/2168167/0/en/Chinook-Receives-Rare-Pediatric-Disease-Designation-from-U-S-Food-and-Drug-Administration-for-CHK-336-for-Treatment-of-Primary-Hyperoxaluria.html

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FDA Grants Rare Pediatric Disease Designation to ONC201 ...

(3 days ago) The Rare Pediatric Disease Priority Review Voucher program, which was created as part of the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012, is intended to incentivize the development of new therapies for rare pediatric diseases.

https://oncoceutics.com/fda-grants-rare-pediatric-disease-designation-to-onc201-for-the-treatment-of-h3-k27m-mutant-glioma/

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Butterfield Introduces Bill to Support Children with ...

(3 days ago) The Creating Hope Act incentivizes the pharmaceutical industry to develop drugs for children with cancer and other life-threatening illnesses through the rare pediatric disease priority review voucher program at the U.S. Food and Drug Administration (FDA). Pediatric cancer remains the number one disease that leads to the death of American children.

https://butterfield.house.gov/media-center/press-releases/butterfield-introduces-bill-to-support-children-with-cancer-and-other

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