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