Search Results for rare pediatric disease

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Have PRVs Incentivized New Rare or Neglected Disease Research? Experts Discuss By Zachary Brennan - Published 08 December 2017

The priority review voucher (PRV) programs, created by Congress with an eye to incentivizing the development of new rare pediatric and neglected tropical disease drugs, have so far rewarded a wide range of small and large biopharma companies and projects, though whether PRVs have actually spurred new research remains a question.

Categories: News, US, FDA, Biologics and biotechnology, Drugs, Regulatory intelligence, Regulatory strategy

Tags: priority review voucher, neglected tropical disease, rare pediatric disease

More Efficient Rare Pediatric Drug Development: FDA Drafts Guidance By Zachary Brennan - Published 06 December 2017

The US Food and Drug Administration (FDA) on Wednesday released draft guidance that could speed the development of treatments for rare pediatric disease drugs.

Categories: News, US, EMA, FDA, Clinical, Drugs

Tags: rare pediatric disease, Gaucher, FDA draft guidance

EMA and FDA Look to Facilitate Development of Gaucher Disease Treatments By Zachary Brennan - Published 03 July 2017

The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) on Monday released a new strategy document to aid in the development of new treatments for Gaucher disease, a rare lysosomal storage disorder.

Categories: News, Europe, US, EMA, FDA, Biologics and biotechnology, Clinical, Drugs, Government affairs, Regulatory intelligence, Regulatory strategy, Submission and registration

Tags: EMA and FDA, Gaucher disease, rare pediatric disease

Priority Review Voucher Fees to Decline in FY 2017 By Zachary Brennan - Published 29 September 2016

The US Food and Drug Administration (FDA) on Thursday unveiled the new user fee rates for the tropical disease and rare pediatric disease priority review voucher (PRV) programs. The additional fees necessary to use the vouchers for both programs are set to decline by about $20,000 when compared to last year.

Categories: News, US, FDA, Biologics and biotechnology, Clinical, Drugs

Tags: priority review vouchers, PRV, rare pediatric disease voucher, golden ticket, tropical disease priority review voucher

Sanofi Redeems $245M Priority Review Voucher for Type 2 Diabetes Treatment By Zachary Brennan - Published 22 February 2016

The US Food and Drug Administration (FDA) accepted for review the New Drug Application (NDA) for Sanofi’s investigational type 2 diabetes treatment on Monday and granted the use of another Priority Review Voucher (PRV), which speeds up FDA’s decision by four months.

Categories: News, US, FDA, Biologics and biotechnology, Compliance, Drugs, Due Diligence, Government affairs, Regulatory intelligence, Regulatory strategy, Submission and registration

Tags: priority review voucher, PRV, tropical disease, rare pediatric disease, orphan drugs

FDA Raises Fee Rate for Rare Pediatric Disease Priority Reviews for FY2016 By Zachary Brennan - Published 25 September 2015

The US Food and Drug Administration (FDA) on Friday announced that it would increase the rare pediatric disease priority review voucher fee rate for FY 2016 by about $200,000.

Categories: News, US, CBER, CDER, Combination products, Drugs, Regulatory intelligence, Regulatory strategy, Reimbursement, Research and development, Submission and registration

Tags: priority review vouchers, rare pediatric disease, review voucher program, Advancing Hope Act of 2015, Cholbam

Regulatory Explainer: Everything You Need to Know About FDA’s Priority Review Vouchers By Alexander Gaffney, RAC, Michael Mezher, Zachary Brennan - Published 29 November 2017

Since 2007, the US Food and Drug Administration (FDA) has issued a handful of special "priority review" vouchers which allow its recipient to expedite the review of any one of its new drug products. What are these vouchers, why is FDA issuing them and what benefit might they have for society? Find out in our latest Regulatory Explainer on the Priority Review Voucher system.

Categories: News, US, CDER, Biologics and biotechnology, Drugs, Regulatory strategy, Research and development, Submission and registration

Tags: Priority Review Voucher, PRV, Neglected Tropical Disease, Neglected Tropical Disease Priority Review Voucher, Rare Pediatric Disease Voucher, Rare Pediatric Disease, FDASIA, FDAAA, Voucher, FDA Voucher, Guidance, AbbVie, United Therapeutics, Praluent

Bill Would Make Permanent FDA's Rare Pediatric Voucher Program By Alexander Gaffney, RAC - Published 24 March 2015

New legislation introduced this week would reauthorize the US Food and Drug Administration's (FDA) rare pediatric disease priority review voucher program, which is currently set to end after triggering a sunset clause in its authorizing statute.

Categories: News, US, CDER, Biologics and biotechnology, Drugs, Regulatory strategy

Tags: Priority Review Voucher, Advancing Hope Act, Rare Pediatric Disease Priority Review Voucher

Pediatric Priority Review Voucher Program Set to End After FDA Approves New Drug By Alexander Gaffney, RAC - Published 18 March 2015

The US Food and Drug Administration's (FDA) rare pediatric disease priority review voucher program is slated to end in one year, after the agency awarded its third-ever rare pediatric voucher, thereby triggering a little-known provision in the voucher program.

Categories: News, US, CDER, Drugs, Regulatory strategy

Tags: Priority Review Voucher, PRV, Asklepion, Retrophin, Rare Pediatric Disease Priority Review Voucher

With New Data in Hand, Could Sanofi and Regeneron Use a Regulatory Shortcut? By Alexander Gaffney, RAC - Published 16 March 2015

A new drug being co-developed by drugmakers Sanofi and Regeneron could, according to new data, dramatically decrease low-density lipoprotein (LDL) cholesterol in patients and lessen cardiac events. But it's a reduction in something else that could be most important for Sanofi and Regeneron: the time it might take the US Food and Drug Administration (FDA) to review the new drug, Praluent.

Categories: News, US, CDER, Biologics and biotechnology, Regulatory strategy, Submission and registration

Tags: Sanofi, Regeneron, Praluent, Priority Review Voucher, Rare Pediatric Disease Priority Review Voucher

For Second Time Ever, FDA Awards Special Voucher Meant to Accelerate Drug Reviews By Alexander Gaffney, RAC - Published 11 March 2015

Drugmaker United Therapeutics has become just the second company in US history to obtain a new type of voucher which allows a company to potentially get its drug approved by the US Food and Drug Administration (FDA) in 40% less time than it normally takes.

Categories: News, US, CDER, Biologics and biotechnology, Drugs

Tags: PRV, Priority Review Voucher, Rare Pediatric Disease Priority Review Voucher, Voucher

FDA Explains How its New Incentive Program for Rare Pediatric Disease Treatments Works By Alexander Gaffney, RAC - Published 18 November 2014

The US Food and Drug Administration (FDA) has released a long-anticipated guidance document intended to explain how a new incentive program known as the Rare Pediatric Disease Priority Review Voucher system works.

Categories: News, US, FDA, Biologics and biotechnology, Drugs

Tags: Rare Pediatric Disease Priority Review Voucher, FDASIA, Voucher, Rare Pediatric Disease Voucher, Priority Review Voucher, Guidance, Draft Guidance