Rare Pediatric Disease Voucher

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

(22 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

(24 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 ...

(4 months 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/2020/09/15/2020-20320/issuance-of-priority-review-voucher-rare-pediatric-disease-product

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Rare Pediatric Disease Priority Review Voucher Program ...

(25 days ago) Whether or not sponsors receive rare pediatric disease designation, they should include a request for a rare pediatric disease voucher in a cover letter to their NDA/BLA submission, if they are...

https://www.fda.gov/drugs/development-resources/rare-pediatric-disease-priority-review-voucher-program-section-529

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FDA updates rare pediatric disease draft guidance

(16 days ago) Under this program, a drug company that receives an approval for a drug or biologic for a rare pediatric disease may qualify for a voucher that can be redeemed to receive a priority review of a...

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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Rare Pediatric Disease Priority Review Vouchers

(24 days ago) 256 populations with the rare pediatric disease, the applicant will still be eligible for a voucher if the 257 approved use includes pediatric use, as described in Question 3 and 4. If the ...

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

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

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

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

(6 days ago) The rare pediatric voucher system is closely modeled off the tropical disease voucher system, with several exceptions. Perhaps the most notable difference at the time of the pediatric voucher's initial passing was its relative ease of use.

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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Rare Diseases | Regulation | Pink Sheet

(1 days ago) The voucher program was extended to rare pediatric diseases in the FDA Safety and Innovation Act of 2012 and, most recently, to medical countermeasures in the 21 st Century Cures Act, which was signed into law in December 2016. Ridley asserts that sales prices were to were too high in 2015 and 2016 given the supply of rare pediatric vouchers.

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

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

(7 days ago) Under section 529 of the FD&C Act (21 U.S.C. 360ff), 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 RINVOQ (upadacitnib) approved August 16, 2019, meets the redemption criteria.

https://www.federalregister.gov/documents/2019/09/06/2019-19298/notice-of-approval-of-product-under-voucher-rare-pediatric-disease-priority-review-voucher

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Issuance of Priority Review Voucher; Rare Pediatric ...

(8 days ago) FDA is announcing the issuance of a priority review voucher to the sponsor of an approved rare pediatric disease product application. 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 ...

https://www.federalregister.gov/documents/2019/03/29/2019-06138/issuance-of-priority-review-voucher-rare-pediatric-disease-product

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

(6 days ago) (202) 512-4800 [email protected] 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.”

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

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Versantis Receives FDA Rare Pediatric Disease Designation ...

(3 months ago) The FDA grants rare pediatric disease designation for serious or life-threatening diseases primarily affecting children from birth to 18 years and affecting fewer than 200,000 people in the USA.

https://www.businesswire.com/news/home/20201018005042/en/Versantis-Receives-FDA-Rare-Pediatric-Disease-Designation-for-VS-01-for-the-Treatment-of-Urea-Cycle-Disorders

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Home - Priority Review Vouchers

(1 days ago) The voucher program is intended to reduce two types of inefficiency. First, the voucher program motivates more treatments for neglected diseases, rare pediatric diseases, and medical countermeasures.

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

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

(5 days ago) December 22, 2020 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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Notice of Approval of Product Under Voucher: Rare ...

(10 days ago) Under section 529 of the FD&C Act (21 U.S.C. 360ff), 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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What happened to the value of priority review vouchers ...

(6 days ago) The pediatric voucher program included several changes, including the ability for the pediatric treatment developer to ask the FDA in advance whether the disease qualifies as a rare, pediatric disease. The marketplace for these PRVs should continue to become more competitive with additional vouchers being issued, and prices will be more closely ...

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

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Rhythm Pharmaceuticals Announces Sale of Priority Review ...

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

(7 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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Priority review - Wikipedia

(1 days ago) Section 529 extends the voucher program to rare pediatric diseases, but only on a trial basis. After the third voucher is awarded, the Comptroller General of the United States is to conduct a study on the effectiveness of the pediatric priority review voucher program. The pediatric voucher program includes changes to the voucher program.

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

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Federal Register :: Fee for Using a Rare Pediatric Disease ...

(7 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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Bill Proposes Continuation Of FDA Rare Pediatric Disease ...

(6 days ago) Historical Incentive for Pharmaceutical Companies 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://www.mondaq.com/unitedstates/food-and-drugs-law/995940/bill-proposes-continuation-of-fda-rare-pediatric-disease-priority-review-vouchers

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Pediatric Rare Disease Voucher Program Extended

(5 days ago) October 9, 2020 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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The FDA Is Ending Its Rare Pediatric Disease Priority ...

(2 days ago) Between 2010 and 2018, 33.8 percent of the 402 orphan indications approved by the FDA were approved for children only or targeted pediatric diseases. 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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Best Travel Voucher Rare Pediatric Disease Voucher ...

(5 months ago) (2 days ago) Under the FDA's rare pediatric disease designation program, the FDA may grant a priority review voucher to a sponsor who receives a product approval for a "rare pediatric disease," which is defined as a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years and affects fewer than 200,000 people in the...

https://fireboxvoucher.com/rare-pediatric-disease-voucher

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Rare Pediatric Disease Designation Requests Skyrocket ...

(2 days ago) Congress should give FDA more flexibility to administer orphan designations, such as the ability to revoke designations later if the disease prevalence grows beyond the rare disease threshold. rare pediatric disease voucher Program Gains Temporary Extension From House Committee

https://pink.pharmaintelligence.informa.com/PS142924/Rare-Pediatric-Disease-Designation-Requests-Skyrocket-Ahead-Of-Voucher-Programs-Sunset

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FDA Grants 3 Rare Pediatric Disease Designations to STAT3 ...

(20 days ago) “The approval of these 3 rare pediatric disease designations is a reminder of just how important our efforts are to potentially help children with brain tumors.” ... These vouchers are issued ...

https://www.onclive.com/view/fda-grants-3-rare-pediatric-disease-designations-to-stat3-inhibitor-wp1066

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Short Sunsets for Priority Review Voucher Programs May ...

(10 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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Marinus Pharmaceuticals Receives Rare Pediatric Disease ...

(5 months ago) The current program for RPD Designation will expire after September 30, 2020 unless Congress renews the program; however, a drug designated for a rare pediatric disease on that date can still ...

https://www.businesswire.com/news/home/20200730005259/en/Marinus-Pharmaceuticals-Receives-Rare-Pediatric-Disease-Designation

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IMR-687 Receives FDA Fast Track & Rare Pediatric Disease ...

(6 days ago) About Rare Pediatric Disease Designation Under the FDA’s Rare Pediatric Disease (RPD) program, a sponsor who receives marketing approval for a product with an RPD designation may be eligible for a voucher that can be redeemed to obtain priority review for any subsequent marketing application. The FDA defines a “rare pediatric disease” as ...

https://thalassaemia.org.cy/news/imr-687-receives-fda-fast-track-rare-pediatric-disease-designations-for-the-treatment-of-%ce%b2-%cf%84halassaemia/

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Moleculin Announces FDA Approves 3 Rare Pediatric Disease ...

(10 days ago) "The approval of these three Rare Pediatric Disease designations is a reminder of just how important our efforts are to potentially help children with brain tumors." ... These vouchers are issued ...

https://www.prnewswire.com/news-releases/moleculin-announces-fda-approves-3-rare-pediatric-disease-designations-for-wp1066-301182314.html

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X4 Pharmaceuticals Receives Rare Pediatric Disease ...

(6 days ago) Under the RPD program, a sponsor who receives an approval for a drug for a “rare pediatric disease” and a Fast Track designation may qualify for a voucher that can be redeemed to receive a priority review by the FDA for any subsequent marketing application for a different product. Such a voucher is transferrable and may be sold.

https://apnews.com/press-release/globenewswire-mobile/business-corporate-news-drug-trials-pharmaceutical-manufacturing-health-care-industry-960d23654e331ec0a884fa025e5f263c

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Rare Pediatric Disease Voucher Program Gains Temporary ...

(2 months ago) In part because assessment costs plummeted in FY 2019, the FY 2021 fee to redeem a priority review voucher will be 37% less than the previous year. Rare Pediatric Disease Designation Requests Skyrocket Ahead Of Voucher Program’s Sunset

https://pink.pharmaintelligence.informa.com/PS142881/Rare-Pediatric-Disease-Voucher-Program-Gains-Temporary-Extension-From-House-Committee

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U.S. FDA grants rare pediatric disease designation to ...

(3 months ago) "This rare pediatric disease designation for AB-G023 shows continued recognition by the FDA of the importance of identifying an improved treatment for CHI patients and their parents," said Pawel Fludzinski, CEO and President of AmideBio. "Amidebio's AB-G023 has the potential to be more readily administered, including for use in pumps."

https://www.biospace.com/article/releases/u-s-fda-grants-rare-pediatric-disease-designation-to-amidebio-s-glucagon-analog-for-the-treatment-of-congenital-hyperinsulinism/

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Priority Review Vouchers for rare pediatric diseases ...

(8 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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Anavex Life Sciences Receives Rare Pediatric Disease ...

(1 months ago) The RPD designation provides priority review by the FDA to encourage treatments for rare pediatric diseases. Under the RPD program, a sponsor who receives an approval for a drug for a “rare pediatric disease” may qualify for a voucher that can be redeemed to receive a priority review by the FDA of a subsequent marketing application for a ...

https://www.anavex.com/anavex-life-sciences-receives-rare-pediatric-disease-designation-from-fda-for-anavex2-73-blarcamesine-for-the-treatment-of-rett-syndrome/

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New Cancer Drug, Three More for Rare Pediatric Diseases ...

(9 days ago) Rhythm Pharmaceuticals earned a voucher for the first-ever FDA approved therapy for rare genetic diseases of obesity. Im cirvee (setmelanotide) will be used for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1 ...

https://www.kidsvcancer.org/new-cancer-drug-three-more-for-rare-pediatric-diseases-approved-and-a-voucher-sells-for-95-million/

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Rare Pediatric Priority Review Voucher Program Seems ...

(9 days ago) Pediatric Rare Disease Voucher Program Faces Expiration 10 Sep 2016. Pink Sheet. Review Voucher Program For Rare Pediatric Diseases Should Not Be Reauthorized, FDA Says 02 Mar 2016. Topics. Subjects Rare Diseases Review Pathway ...

https://pink.pharmaintelligence.informa.com/PS142639/Rare-Pediatric-Priority-Review-Voucher-Program-Seems-Headed-For-Short-Term-Renewal

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Taysha Gene Therapies Receives Rare Pediatric Disease and ...

(1 months 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/

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Praxis Precision Medicines (PRAX) Receives Rare Pediatric ...

(6 days ago) Under the FDA’s rare pediatric disease and voucher programs, subject to FDA approval of a product with such designation, a company may be eligible to receive a priority review voucher that can ...

https://www.streetinsider.com/Corporate+News/Praxis+Precision+Medicines+%28PRAX%29+Receives+Rare+Pediatric+Disease+and+Orphan+Drug+Designations+for+Severe+Pediatric+Epilepsy+Programs/17795251.html

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Editas Medicine Receives Rare Pediatric Disease ...

(9 days ago) Pediatric diseases recognized as “rare” affect under 200,000 people in the United States. Under the FDA’s Rare Pediatric Disease Designation and Voucher Programs, a sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may be eligible for a voucher that can be redeemed to receive priority review of a ...

https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-receives-rare-pediatric-disease-designation-edit

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Taysha Gene Therapies Receives Rare Pediatric Disease ...

(2 months 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/20201014005319/en/Taysha-Gene-Therapies-Receives-Rare-Pediatric-Disease-Designation-and-Orphan-Drug-Designation-for-TSHA-102-as-a-Treatment-for-Rett-Syndrome

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Scholar Rock's experimental SMA therapy nabs speedy review ...

(8 days ago) The FDA has handed Scholar Rock a rare pediatric disease designation voucher, which can allow the biotech a speedy review of its leading of spinal muscular atrophy (SMA) hopeful SRK-015.

https://www.fiercebiotech.com/biotech/scholar-rock-s-experimental-sma-therapy-nabs-speedy-review-voucher

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

(10 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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Priority Review Vouchers Are A Big Carrot For Hungry ...

(10 days ago) Rare pediatric diseases are defined by FDA as diseases that primary affect individuals age birth to 18 years (interpreted in the FDA guidance on rare pediatric vouchers as greater than 50% of those affected in US in the 0 – 18 years age range) and affects fewer than 200,000 persons in the US (or a disease that affects larger numbers of ...

https://camargopharma.com/resources/blog/priority-review-vouchers-a-big-carrot-for-hungry-companies/

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LogicBio Therapeutics Receives Rare Pediatric Disease ...

(9 days ago) The FDA grants rare pediatric disease designation for serious and life-threatening diseases that primarily affect children through age 18 and affect fewer than 200,000 people in the United States. Under the FDA's rare pediatric disease priority review voucher program, a sponsor who receives approval of a new drug application (NDA) ...

https://investor.logicbio.com/news-releases/news-release-details/logicbio-therapeutics-receives-rare-pediatric-disease

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