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Orphan Drugs And Rare Diseases: 22% of FDA CDER 2014 Approvals Are Personalized Medicines

January 29, 2015

The Personalized Medicine Coalition’s (PMC) mission is:

“ … to educate federal and state policymakers and private sector health care leaders about personalized medicine, helping them understand the science, the issues and what is needed for the positive evolution of personalized medicine – as critically important.

Biopharmaceutical companies developing orphan drugs for rare diseases, are working with diagnostic companies or diagnostic areas within their companies, to establish companion diagnostics and biomarkers. The purpose for developing diagnostics and biomarkers is to help identify patients who have a gene or genes specific to an orphan drug in development and who are likely to benefit from treatment with the drug. It is a method of establishing patient groups in which an orphan drug will be effective – personalized medicine.

In January 2015, FDA’s CDER (Center for Drug Evaluation and Research) publishes its fourth annual report, “Novel New Drugs Summary”, for calendar year 2014. The report identifies the approved 41 novel new drugs, approved as New Molecular Entities (NMEs) under New Drug Applications (NDAs) or as new therapeutic biologics under Biologics License Applications (BLAs).

PMC reviews FDA CDER’s 41 novel new drugs and classifies 9 of them, approximately 22% as personalized medicines. PMC defined personalized medicines as those products “for which the label includes reference to specific biological markers, identified by diagnostic tools, that help guide decisions and/or procedures for the product’s use in individual patients.”

The chart below identifies the 9 PMC personalized medicines:

Row Num Trade Name/ Biomarker Sponsor Company FDA ODD Approval Date Indication
1 Lynparza (Olaparib)/ BRCA biomarker AstraZeneca 12.19.14 Advanced Ovarian Cancer
2 Vimizin (Elosulfase Alpha)/ Type A or B biomarker BioMarin Pharmaceutical 02.14.14 MPS IV A (Morquio A Syndrome)
3 Cyrazma (Ramucirumab)/ EGFR or ALK biomarker Eli Lilly & Co 04.21.14 & 11.05.14 Advanced Gastric/ Gastro-Esophageal Junction Adenocarcinoma/ NSCLC
4 Zykadia (Ceritinib)/ ALK biomarker Novartis 04.29.14 NSCLC
5 Beleodaq (Belinostat)/ UGT1A1 biomarker Spectrum Pharmaceuticals 07.03.14 Peripheral T-Cell Lymphoma (PTCL)
6 Cerdelga (Eliglustat)/ CYP2D6 biomarker Genzyme 08.09.14 Gaucher Disease Type 1
7 Harvoni (Ledipasvir + Sofosbuvir)/ Hepatitis C Genotype 1 biomarker Gilead Sciences No ODD, but FDA BTD ** approval 10.10.14 Chronic Hepatitis C
8 Viekira Pak (Ombitasvir, Paritaprevir, Ritonavir)/ Hepatitis C Genotype 1 biomarker AbbVie No ODD, but FDA BTD ** approval 12.19.14 Chronic Hepatitis C
9 Blincyto (Blinatumomab)/ Philadelphia Chromosome biomarker Amgen 12.03.14 B-cell precursor acute lymphoblastic leukemia (ALL)

Source of Data: FDA ODD Database + PMC Report.

## = FDA Breakthrough Therapy Designation (BTD).

Please Note: “Nessler Cylinders” by Panek (Own work) [GFDL or CC-BY-3.0 | via Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

  1. Manfred Roesner permalink

    I read your article with great interest. Having followed the 2014 FDA new drug approvals, I wonder, if not pembrolizumab and nivolumab should be added to the 2014 PMs as well? Both melanoma drugs require the presence of the BRAF V600 mutation according to the approval summary. Thanks for checking and regards.

    • Hi Manfred – You are correct about Keytruda (Pembrolizumab) as per the PI (Package Insert) at URL, under the “Indications & Usage Section”, it says ” …. if BRAF V600 mutation positive, a BRAF indicator”. Also, for Opdivo (Nivolumab), per the FDA Press Release at URL, it refers to ” … for melanoma patients whose tumors express a gene mutation called BRAF V600 ….”.

      PMC defined personalized medicines as those products “for which the label includes reference to specific biological markers, identified by diagnostic tools, that help guide decisions and/or procedures for the product’s use in individual patients.”

      In conclusion, I agree with you 100% and thanks for bringing up this fact. Kind Regards, Ann

  2. NOTE, THE SOURCE DATA USED IS TERRIBLY FLAWED, in terms of reporting the total number of new (bio)pharmaceuticals approved in 2014. You fell into the trap laid every recent year by CDER – using its approvals review information and presuming t is complete for FDA. But this reporting totally ignores the biologics/BLAs approved by CBER! It only includes CDER-regulated products (both drugs and biologics). Your source list is in no way comprehensive concerning ‘new’ FDA 2014 approvals!

    See my running list of biopharmaceutical approvals at New products, all or most also clearly containing new molecular entities, approved with full BLAs by CBER in 2014 (what you missed considering) include:
    1) recombinant porcine (pig) Factor VIII, Obizur, from Baxter*
    2) recombinant glucagon-like peptide-1 (GLP-1), Trulicity, from Lilly
    3) recombinant complement C1 esterase inhibitor, Rhucin, expressed by transgenic rabbits (can’t get more novel than this), from Salix Pharmaceuticals, Ltd. (and Pharming Group NV)
    4) recombinant Factor VIII, Eloctate, from Biogen-Idec
    5) recombinant Factor IX-Fc fusion protein, Alprolix, from Biogen-Idec
    6) recombinant human papiillovirus (HPV) vaccine, Gardasil 9, from Merck
    7) meningococcal group B vaccine, Trumemba, from Pfizer
    8) hyaluronic acid, cross-linked, Monovisc, from Anika Therapeutics
    9) a new combination — Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase – HYQVIA (Gammagard plus Hylenex) from Baxter

    With a high proportion of these approved with orphan status, perhaps the % of personalized medicine approvals in 2014 might further increase, if you recalculate using all relevant 2014 FDA approvals.

    Thank you.

    Ronald A. Rader
    Author/Publisher:  BIOPHARMA:  Biopharmaceutical Products in the U.S. and European Markets
    Biotechnology Information Institute
    1700 Rockville Pike, Suite 400
    Rockville, MD 20852

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