CATEGORIES: Research
what is fast track

What Is Fast Track?

by | May 29, 2019 | 2 comments

Acceleronpharnext

 

THE FDA HAS AWARDED FAST TRACK DESIGNATION TO TWO DRUGS CURRENTLY IN DEVELOPMENT FOR CMT:

ACCELERON’S ACE-083 AND PHARNEXT’S PXT3003

Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions.

Fast Track designation must be requested by the drug company. The request can be initiated at any time during the drug development process. FDA will review the request and make a decision within sixty days based on whether the drug fills an unmet medical need in a serious condition.

Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.

SOURCE: U.S. FOOD AND DRUG ADMINISTRATION www.fda.gov/ForPatients/Approvals/Fast/ucm405399.htm

Acceleron ACE-083

HNF partner, Acceleron Pharma, announced in November, 2018, that the FDA had granted Fast Track status to ACE-083, an injected myostatin drug intended to increase muscle mass and strength. Acceleron announced the exciting, preliminary positive results of the part 1 of the phase 2 clinical trial in January, 2019.

“We’re pleased that the FDA has granted this designation for ACE-083,” said Robert K. Zeldin, M.D., Chief Medical Officer of Acceleron. “Patients with CMT currently have no approved therapies. To date, results from our Phase 2 trials have shown that patients treated with ACE-083 experience robust increases in muscle volume. If our ongoing clinical studies show that ACE-083 also improves functional outcomes and confirm the favorable safety profile observed thus far, the Fast Track process could help us work with the FDA to deliver it to patients as quickly as possible.”

Preliminary results from part 2 of the trials are expected by year end 2019.

Pharnext PXT3003

HNF partner, Pharnext, announced in February, 2019, that they had been granted Fast Track designation for the development of PXT3003 for the treatment of patients with CharcotMarie-Tooth disease Type 1A (CMT1A). PXT3003 is a novel synergistic combination of baclofen, naltrexone and sorbitol, formulated as an oral solution that is given twice a day.

“We are pleased to receive Fast Track designation for PXT3003 in CMT1A” said Daniel Cohen, M.D., Ph.D., cofounder and Chief Executive Officer of Pharnext. “CMT1A is a rare, chronic neuropathy that affects at least 125,000 people across the U.S. and Europe. There is currently no approved treatment for this disease. We look forward to continuing our productive dialogue with the FDA to rapidly progress towards a U.S. New Drug Application for PXT3003, to deliver this therapy to patients as quickly as possible.”

Learn more on this topic

Related Blog Posts

Building Awareness on CMT and Supporting the Patient Community – HNF Announces Support from Pharnext

HNFpharnext recently entered into a partnership with the French biopharmaceutical company, Pharnext, to help raise awareness of Charcot-Marie-Tooth (CMT) disease and support the CMT patient community through several initiatives. Building awareness is key! Pharnext’s support will assist HNF in distributing HNF’s CMT Update quarterly newsletter, enhancing the Global Registry for Inherited Neuropathies (GRIN), setting up activities for CMT September Awareness Month in the US and strengthening the CMT Inspire Community.

Quality of Life and CMT Research

Did you know that 95% of clinical trials fail? There are multiple causes, most related to efficacy or safety, which obviously can be harmful and risky for patients. The risk-reward of enrolling in trials is a judgment call based on the devastating effects of disease related to quality of life (QoL) or life-threatening disease. With CMT, the risk-reward is more of a challenging question for many, as CMT in most cases is non-fatal.

Help us answer questions that your doctors and the CMT Research Community aren’t too sure about.

Did you know that you can become part of a community in therapy development and further research for all forms of CMT and inherited neuropathies? The mission of the Global Registry for Inherited neuropathies (GRIN) is to collect clinical and genetic information from patients with ALL forms of Charcot-Marie-Tooth (CMT) and other related rare and ultra rare inherited neuropathies.

Hot off the press

It seems like almost weekly there is another new publication on CMT with interesting basic biology. While an earlier “Hot off the press” highlighted the work of Cherry and co-workers at the University of Texas Southwestern Medical Center who showed show that neurons lacking a gene for rab7 result in neuropathy.

New Study on CMT Type 2B

If we are to learn more about CMT and the effectiveness of rehabilitation it is worth asking the patient and their caregiver. A recent Italian study by Padua et al recently described a survey of CMT patients and caregivers and their perspectives and perceptions of rehabilitation efficacy and needs.

Allison Moore is going to be published!

HNF’s CSO (Chief Scientific Officer), Sean Ekins wrote a blog about his work with Allison Moore and her two “fighter Mom” friends Lori Sames and Jill Wood. He named his blog: “Rare disease heroes – Extraordinary collaborators we should be listening too.” Sean helped my friends and I write a paper called: “Multifaceted roles of ultra rare and rare disease patients/parents in drug discovery.” YES, Allison Moore is going to be published! It will be in Drug Discovery Today, soon. The link to the reprint is below.

HNF-WVU-NIOSH Study update:

We are pleased to update our readers on the progress of the WVU-NIOSH study, “A Bi-Directional Translational Model of Exercise Training in the Treatment of Charcot-Marie-Tooth Disease”

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2 Comments

  1. Alison Connor

    I have cmt1 also 4of my children my aunt’s cousins my children attend a neurologist and have orthotics. for their shoes I am suffering with pains in my arm shooting down from my shoulder to hand and my hand is locking alot more a y advice and my man and me are suffering with hot feet thanks Alison

    Reply
  2. Lisamarie Fleenor

    Awesome!

    Reply

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