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Filtering on: "topics orphan products"

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FDA's Frey Reflects on Final Year of PDUFA V By Michael Mezher - Published 06 December 2017

As the year comes to a close, Patrick Frey, senior advisor to the director of the Office of New Drugs (OND) at the Center for Drug Evaluation and Research (CDER), looked back at FDA's performance over the final year of the fifth iteration of the Prescription Drug User Fee Act (PDUFA V) and what's to come in PDUFA VI.

Categories: News, US, CDER, Biologics and biotechnology, Communication, Drugs, Orphan products, Submission and registration

Tags: PDUFA, NDA, BLA, Orphan Drugs, Approvals

As Transparency on Priority Review Vouchers Fades, Prices Level Off By Zachary Brennan - Published 29 November 2017

In the early days of the priority review voucher (PRV) programs, back in 2014 and 2015, companies publicly disclosed who they sold the PRVs to, how much they cost and which products they were eventually used to speed the review of.

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

Tags: priority review vouchers, transparency, PRV prices

FDA Analyst Counters Critiques of Orphan Drug Act By Michael Mezher - Published 18 October 2017

While recent reports have claimed that drugmakers are manipulating the incentives provided by the Orphan Drug Act, Mike Lanthier, an operations research analyst at the US Food and Drug Administration (FDA), says that in most cases, the act is working as intended.

Categories: News, FDA, Biologics and biotechnology, Drugs, Orphan products

Tags: Orphan Drugs, Orphan Drug Act

FDA Officials Offer Advice on Efficient Orphan Drug Development By Michael Mezher - Published 17 October 2017

A group of top US Food and Drug Administration (FDA) officials shared insights on how drugmakers can be more efficient when developing treatments for rare diseases, according to comments on Tuesday at the National Organization for Rare Disorders' (NORD) Summit in Washington, DC.

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

Protalex Wins $403K Grant from FDA Office of Orphan Products Development By Zachary Brennan - Published 18 August 2017

Clinical-stage biopharma Protalex, Inc. on Thursday said it has been awarded a $403,000 grant from the US Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) to support future clinical development activity of PRTX-100 as a treatment for immune thrombocytopenia (ITP).

Categories: News, US, FDA, Clinical, Drugs, Orphan products

Tags: OOPD, orphan drug, Protalex

10 Years in, EU Pediatric-Use Marketing Authorization Called a 'Disappointment' By Zachary Brennan - Published 06 July 2017

Biopharmaceutical companies and regulators across Europe have said that in the 10 years since the Pediatric Regulation took effect, some pediatric therapeutic areas have seen progress, though the pediatric-use marketing authorization (PUMA) has not lived up to expectations.

Categories: News, Europe, EC, EMA, MHRA, Biologics and biotechnology, Drugs, Orphan products, Regulatory intelligence, Regulatory strategy

Tags: pediatric-use marketing authorisation, EMA and PUMA, pediatric drugs

FDA Plots Elimination of the Orphan Drug Designation Request Backlog By Zachary Brennan - Published 29 June 2017

With a flood of new orphan drug designation requests, the US Food and Drug Administration (FDA) on Thursday released its new plan to eliminate the backlog of requests in 90 days and to respond to 100% of all new orphan drug designation requests within 90 days.

Categories: News, US, FDA, Drugs, Government affairs, Orphan products, Regulatory intelligence, Regulatory strategy

Tags: orphan drug, orphan medical product, orphan designation, orphan designation backlog

FDA Commissioner Vows to Eliminate Backlog of Orphan Drug Designation Requests By Michael Mezher - Published 20 June 2017

At a Senate hearing on President Donald Trump's FY2018 budget request for the US Food and Drug Administration (FDA), Commissioner Scott Gottlieb vowed to eliminate the backlog of orphan drug designation requests and said he will soon release "modern and risk-based" tools for assessing new treatments, especially for rare diseases and conditions with no effective treatments.

Categories: News, US, FDA, Generic drugs, Orphan products

Tags: FY2018 Budget, Appropriations, Orphan Drug Designations, REMS, Scott Gottlieb

TGA to Move Forward With Orphan Drug Revamp By Michael Mezher - Published 18 April 2017

Australia's pharmaceutical industry is "broadly supportive" of proposed changes to the country's orphan drug program, the Therapeutic Goods Administration (TGA) says.

Categories: News, Oceania, TGA, Biologics and biotechnology, Drugs, Orphan products, Regulatory strategy, Submission and registration

Tags: Orphan Drugs, Australia

House Bill Would Incentivize Drugmakers to 'Repurpose' Drugs for Rare Diseases By Michael Mezher - Published 02 March 2017

Rep. Gus Bilirakis (R-FL) on Monday reintroduced a bill that would grant drugmakers an additional six months exclusivity for repurposing already-approved drugs to treat rare diseases.

Categories: News, US, FDA, Biologics and biotechnology, Drugs, Government affairs, Orphan products, Submission and registration

Tags: OPEN Act, Marketing Exclusivity,

EMA Reflects on its First Decade of Conditional Marketing Authorizations By Michael Mezher - Published 24 January 2017

Ten years after the development of the conditional marketing authorization (CMA) pathway, European Medicines Agency (EMA) on Monday said the program is working as intended, and has led to earlier patient access to 30 drugs, nearly half of which were for orphan indications, and 80% of which target seriously debilitating or life-threatening conditions.

Categories: News, Europe, EMA, Biologics and biotechnology, Clinical, Drugs, Orphan products, Regulatory strategy, Submission and registration

Tags: Conditional Marketing Authorisation, CMA

European Commission Clears up Questions on Orphan Drug Regulation By Michael Mezher - Published 05 January 2017

Following a public consultation in November 2015, the European Commission (EC) has released a new communication intended to clarify some lingering questions about the EU's Orphan Regulation.

Categories: News, Europe, EC, EMA, Biologics and biotechnology, Drugs, Orphan products

Tags: Orphan Regulation, Orphan Drugs, Orphan Designation

FDA’s Office of New Drugs Director to Retire By Zachary Brennan - Published 05 December 2016

After nearly 25 years of service at the US Food and Drug Administration (FDA), John Jenkins, MD, Director of the Office of New Drugs (OND), will retire from the federal government on 6 January 2017.

Categories: News, US, CDER, Biologics and biotechnology, Drugs, Government affairs, Orphan products, Regulatory intelligence, Regulatory strategy

Tags: John Jenkins, Office of New Drugs, OND, CDER's OND, new drug applications

European Commission to Overhaul Concept of ‘Similar’ Medicines Within Context of Orphan Legislation By Zachary Brennan - Published 29 July 2016

The European Commission on Friday proposed to review the concept of “similar medicinal products” in the context of its orphan legislation as part of a wider effort to adapt the text to technical progress.

Categories: News, Europe, EC, Active pharmaceutical ingredients, Biologics and biotechnology, Drugs, Government affairs, Orphan products

Tags: orphan drug legislation, European Commission, gene therapy, ATMP

Can Health Technology Assessment be Used to Demonstrate Significant Benefit for an Orphan Medicinal Product in the EU? By Richard Phillips, Adriaan Fruijtier, Mauro Placchi - Published 07 July 2016

This article discusses how the conceptual grounds for significant benefit are addressed in the context of the orphan medicinal product regulations in the EU and how techniques used in Health Technology Assessment (HTA) may support the justification for the claim of significant benefit.

Categories: Features, Europe, EC, EMA, Biologics and biotechnology, Drugs, Orphan products, Reimbursement

EMA's 2015 Annual Report: Interest in Scientific Advice Increasing By Michael Mezher - Published 17 May 2016

The European Medicines Agency (EMA) on Tuesday released its Annual Report 2015 touting its performance and key achievements for the year.

Categories: News, Europe, EMA, Biologics and biotechnology, Drugs, Orphan products, Regulatory strategy, Submission and registration

Tags: Guido Rasi, Sir Kent Woods, New active substances, Scientific Advice

Study: Orphan Drugs More Innovative Than Non-Orphan Products By Michael Mezher - Published 23 March 2016

A recent study of new drugs approved by the US Food and Drug Administration (FDA) finds that orphan products are more innovative than their non-orphan counterparts, representing roughly 40% of first-in-class drug approvals between 1983 and 2014.

Categories: News, US, FDA, Biologics and biotechnology, Drugs, Orphan products, Submission and registration

EU High Court Upholds Orphan Exclusivity Ruling By Michael Mezher - Published 23 March 2016

Earlier this month, the EU's top court upheld a major ruling with implications for orphan drug exclusivity by rejecting an appeal by generic drugmaker Teva Pharmaceuticals Europe.

Categories: News, Europe, EC, EMA, Biologics and biotechnology, Drugs, Government affairs, Orphan products, Submission and registration

Tags: Glivec, Gleevec, Tasigna, Teva, Novartis, Market exclusivity

Regulators, Industry Weigh in on Proposed EU Orphan Regulation Changes By Zachary Brennan - Published 07 March 2016

As the number of orphan drug approvals continues to rise in the EU, the European Commission is now considering feedback from European governments, regulators and industry on how changes to five aspects of the Orphan Regulation may further incentivize the development of drugs for rare diseases.

Categories: News, Europe, EC, EMA, MHRA, Drugs, Government affairs, Orphan products

Tags: orphan drugs, Orphan Regulation, EC consultation, rare disease drugs

$2 Million in Funding From FDA for Rare Disease Natural History Studies By Michael Mezher - Published 29 February 2016

The US Food and Drug Administration (FDA) will award $2 million in grants to fund natural history studies for rare diseases.

Categories: News, US, FDA, Orphan products

Orphan Medicines in the EU: A 15-Year Review By Zachary Brennan - Published 29 January 2016

With the 15th anniversary of the adoption of the Orphan Regulation in the EU, the European Commission notes in a new report that this landmark regulation has incentivized the development, approval and marketing of more than 100 rare disease treatments, or orphan drugs.  

Categories: News, Europe, EC, EMA, Biologics and biotechnology, Drugs, Government affairs, Manufacturing, Orphan products, Research and development

Tags: orphan medicines, rare disease drugs, cancer drugs, orphan drug incentives

FDA Approves First Drug to Improve Overall Survival in Liposarcoma By Michael Mezher - Published 28 January 2016

The US Food and Drug Administration (FDA) on Thursday approved a new indication for Eisai's Halaven (eribulin mesylate) to treat liposarcoma, a rare and deadly form of soft tissue sarcoma (STS) that affects only a few thousand patients in the US each year.

Categories: News, US, FDA, Drugs, Orphan products, Submission and registration

Tags: Halaven, erubulin, Liposarcoma

EMA Carries 2014 Momentum, Recommends 39 New Drugs and Sets Orphan Record By Michael Mezher - Published 12 January 2016

In 2015, the European Medicines Agency (EMA) maintained its momentum from 2014, granting 93 positive opinions, 39 of which were for new active substances. The agency also recommended 18 orphan drugs for approval, breaking last year's record of 17.

Categories: News, Europe, EMA, CDER, Biologics and biotechnology, Drugs, Orphan products, Submission and registration

Tags: New active substance, new chemical entity, 2015 approvals, Marketing authorization

Proposed Rule Would Revise Regulations on Fixed-Combination Drugs By Zachary Brennan - Published 22 December 2015

The US Food and Drug Administration (FDA) is proposing a new rule that would apply its regulations on prescription fixed-combination drugs to a variety of nonprescription fixed-combination and co-packaged drugs, as well as to combinations of over-the-counter active ingredients.

Categories: Biologics and biotechnology, Business Skills, Clinical, Combination products, Compliance, Drugs, Ethics, Government affairs, Labeling, Orphan products, Packaging, Preclinical, Project management, Regulatory strategy, Submission and registration

Tags: fixed-combination drugs, FDA rulemaking, proposed FDA rule, OTC drug monographs, nonprescription drug combinations

European Commission to Evaluate EMA’s Fee System By Zachary Brennan - Published 22 December 2015

Late next year, the European Commission will begin reviewing the pharmaceutical company fee system that helps fund the European Medicines Agency (EMA).

Categories: News, Europe, EC, EMA, Clinical, Compliance, Drugs, Due Diligence, Ethics, Government affairs, Manufacturing, Orphan products, Regulatory strategy, Reimbursement, Submission and registration

Tags: EMA, pharmacovigilance, marketing authorization fees in EU, marketing authorisation