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Orphan Drugs And Rare Diseases: Pfizer In The News

September 10, 2014

This Blog Post reviews recent news for Pfizer in the rare disease and orphan drug space on two topics:

•   Deal with Solid Ventures to test potential Duchenne Muscular Dystrophy (DMD) drug

•   FDA Orphan Drug Designation (ODD) for Sickle Cell Disease.

I – Solid Ventures Collaboration For DMD

Solid Ventures is a biotechnology company (Cambridge, Massachusetts) founded in 2013, by a father, Ilan Ganot, after his son, Eytani, is diagnosed with DMD. Mr. Ganot quit his job in London, moved to the Boston area, raised $17 million, and founded Solid Ventures. Eytani’s name translated from Hebrew to English is “solid”, and thus the company’s name. Solid Ventures, a for-profit company, is patient-focused and condition-focused solely on DMD.

With the financial support from JPMorgan (Mr. Ganot’s former employer) and other life sciences venture capitalists, Solid Ventures plans to acquire or license promising projects from other companies or academic laboratories, “move them through the earliest stages of testing with the help of contract research organizations, and ultimately partner or sell them to bigger pharmaceutical companies that can take them to market.”

Pfizer is collaborating with Solid Ventures in a program, “Solid One” :

“ … for pre-clinical research … to test a specific experimental drug in Pfizer’s portfolio for possible use in the treatment of DMD. Solid is leading a study with an expert research partner designed to assess the potential effectiveness of this therapeutic, and aim to make clinical development decisions together with our collaborator late in 2014. Pfizer’s innovation, vision and leadership in the rare disease space is outstanding … In addition, Solid and Pfizer are exploring further ways to collaborate, and continue to work on behalf of every child and family affected by this terrible disease to find a treatment and eventually a cure.”

II – FDA ODD For Sickle Cell Disease

On September 4th, the FDA grants Pfizer an Orphan Drug Designation (OOD) for Sickle Cell Disease (SCD):

Generic Name: 6-((3S,4S)-4-Methyl-1-pyrimidin-2-ylmethyl-pyrrolidin-3-yl-1-(tetrahydro-pyran-4-yl)-1,5-dihydro-pryazolo[3,4-d]pyrimidin-4-one
Trade Name: n/a
Date Designated: 09-04-2014
Orphan Designation: Treatment of sickle cell disease
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Pfizer, Inc. 500 Arcola Road Collegeville, PA 19426 The sponsor address listed is the last reported by the sponsor to OOPD.


On September 15, 2014, the 3rd Annual Sickle Cell Disease Therapeutics Conference will be held in New York City.  The one-day meeting includes presentations from key opinion leaders, clinical-stage companies, patients and families, and healthcare analysts.

Just this week, the NIH produces the first evidence-based report to help clinicians in preventing and treating conditions affecting sickle cell disease.  The report, “Evidence-Based Management of Sickle Cell Disease, Expert Panel Report, 2014” is available on NIH’s website.

Please Note: “Spinning Device To Mix Tissue Culture Medium” by National Cancer Institute [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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