Personalized Medicines: Promise or Not?

Much attention has been given to personalized medicines as a means of transforming healthcare with potentially more effective and better health outcomes, but to what extent is the promise of personalized medicines being realized? DCAT Value Chain Insights takes an inside look.

Personalized medicines approved as new molecular entities
Personalized medicine, which is also called precision or individualized medicine, is an evolving field in which physicians use diagnostic tests to determine which medical treatments will work best for each patient, according to a definition provided the Personalized Medicines Coalition (PMC), an industry advocacy group for personalized medicines. By combining the data from diagnostic tests with an individual’s medical history, circumstances and values, the goal of personalized medicines is to develop targeted treatment and prevention plans.

In evaluating the new molecular entities (NMEs) approved by the US Food and Drug Administration’s Center for Drug Evaluation and Research, PMC defined personalized medicines as those therapeutic products for which the label includes reference to specific biological markers, identified by diagnostic tools, that help guide decisions and/or procedures for their use in individual patients. Using that as a definition, PMC identified that 13 of the 45 NMEs approved in 2015 (see Table I) may be classified as personalized medicines.

Table I: 2015 New Molecular Entities (New Drug Applications (NDAs) and Original Biologics License Applications (BLAs) Approved by the US Food and Drug Administration’s Center for Drug Evaluation and Research Classified as Personalized Medicines.
Company Property name (active ingredient); application type; Indication
Alkermes Aristada (aripiprazole lauroxil) extended-release injection; NDA Schizophrenia
Asklepion Pharmaceuticals/Retrophin Cholbam (cholic acid); NDA Pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for patients with peroxisomal disorders
AstraZeneca Tagrisso (osimertinib); NDA Advanced non-small cell lung cancer in patients whose tumors have a specific epidermal growth factor receptor (EGFR) mutation (T790M) and whose disease has gotten worse after treatment with other EGFR-blocking therapy
Amgen Repatha (evolocumab); BLA For some patients who are unable to get their low-density lipoprotein (LDL) cholesterol under control with current treatment options
Bristol-Myers Squibb Daklinza (daclatasvir); NDA For use with sofosbuvir to treat hepatitis C virus genotype 3 infections
GlaxoSmithKline Nucala (mepolizumab); BLA With other asthma medicines for the maintenance treatment of asthma in patients age 12 years and older
Otsuka Pharmaceutical Rexulti (brexpiprazole); NDA Adults with schizophrenia and as an add-on treatment to an antidepressant medication to treat adults with major depressive disorder
Pfizer Ibrance (palbociclib); NDA Metastatic breast cancer
Roche/Genentech Alecensa (alectinib); NDA Advanced ALK-positive non-small cell lung cancer
Roche/Genentech Cotellic (cobimetinib); NDA In combination with vemurafenib to treat advanced melanoma that has spread to other parts of the body or can’t be removed by surgery, and that has a certain type of abnormal gene (BRAF V600E or V600K mutation)
Sanofi/Regeneron Pharmaceuticals Praluent (alirocumab); BLA For certain patients with high cholesterol
Taiho Oncology Lonsurf (trifluridine and tipiracil) NDA Advanced colorectal cancer
Vertex Pharmaceuticals Orkambi (lumacaftor and ivacaftor); NDA Cystic fibrosis in patients who have two copies of a specific mutation

Retrophin agreed to acquire Chobalm from Asklepion Pharmaceuticals in January 2015 and exercised a purchase agreement in March 2015. Shire acquired NPS Pharmaceuticals in February 2015. Repatha was approved for two indications, one for a rare disease indication, one not. Source: US Food and Drug Administration’s Center for Drug Evaluation and Research and company information.


The Class of 2015: personalized approved as new molecular entities
In looking at the personalized medicines approved in 2015, Amgen and Sanofi both scored NME approvals by the FDA for a new class of anti-cholesterol drugs, human monoclonal antibodies that inhibit proprotein convertase subtilisin/kexin type 9 (PCSK9), a protein that reduces the liver’s ability to remove low-density lipoprotein cholesterol (LDL-C), or “bad” cholesterol, from the blood. Amgen’s Repatha (evolocumab) and Sanofi’s/Regeneron’s Praluent (alirocumab) are pegged as potential blockbusters. Based on estimates for 2019 sales, a recent Thomson Reuters analysis puts potential revenues at Regeneron Pharmaceuticals and Sanofi’s Praluent (alirocumab) at $1.96 billion, and Amgen’s’ evolocumab at nearly $2.0 billion by 2019. In addition to receiving US approval, both drugs have been approved in the European Union.

Pfizer and Otsuka Pharmaceutical also received approval for personalized medicines with blockbuster potential. Otsuka received FDA approval for Rexulti (brexpiprazole), an psychotropic compound discovered by Otsuka and co-developed with Lundbeck, for treating adults with schizophrenia and as an add-on treatment to an antidepressant medication to treat adults with major depressive disorder. The Thomson Reuters analysis estimates potential 2019 sales of $1.09 billion. Pfizer’s Ibrance (palbociclib), a drug to treat advanced (metastatic) breast cancer, is expected to be a strong performer for Pfizer, with the Thomson Reuters analysis offering 2019 sales estimates of $4.6 billion. Ibrance is a kinase inhibitor indicated in combination with letrozole for the treatment of postmenopausal women with estrogen receptor-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced breast cancer as initial endocrine-based therapy for their metastatic disease. Another personalized medicine approved in 2015 with potential blockbuster status is Vertex Pharmaceuticals’ Orkambi (lumacaftor and ivacaftor) for treating cystic fibrosis. 

Other personalized medicines approved in 2015 by the large pharmaceutical companies include: AstraZeneca’s Tagrisso (osimertinib) for treating non-small-cell lung cancer; Bristol-Myers Squibb’s Daklinza (daclastavisr) for treating hepatatis C (genotype 3); GlaxoSmithKline’s Nucala (mepolizumab) for treating asthma;. Roche/Genentech’s Alecensa (alectinib) for treating advanced ALK-positive non-small cell lung cancer and Cotellic (cobimetinib) for treating advanced melanoma.

Of the 13 personalized medicines approved as NMEs in 2015, five are oncology drugs, according to data from the PMC. These drugs account for 35% of the 14 oncology NMEs approved in 2015. In 2015, there were also several personalized medicines that received approval for additional indications, including AstraZeneca’s anticancer drug, Iressa (gefitinib); AbbVie’s Technivie (ombitasvir, paritaprevir, ritonavir, ribavirin) for treating hepatitis C; and two anticancer drug, the PD-1 checkpoint inhibitors, Bristol-Myers Squibb’s Opdivo (nivolumab) and Merck & Co.’s Keytruda (pembrolizumab).

Government initiatives
On governmental level, an important policy initiative is the Precision Medicine Initiative (PMI), which was first announced by President Barack Obama in July 2015. The President’s budget for fiscal year 2016 included $216 million in funding for the initiative for the National Institutes of Health (NIH), the National Cancer Institute (NCI) and the Food and Drug Administration. The Precision Medicine Initiative has both short-term and long-term goals. The short-term goals involve expanding precision medicine in the area of cancer research. Researchers at the NCI hope to use this approach to find new, more effective treatments for various kinds of cancer based on increased knowledge of the genetics and biology of the disease. The long-term goals of the Precision Medicine Initiative focus on bringing precision medicine to all areas of health and healthcare on a large scale. To this end, the NIH plans to launch a study involving a group (cohort) of at least 1 million volunteers from around the United States. Participants will provide genetic data, biological samples, and other information about their health. These data will be used by researchers to study a large range of diseases, with the goals of better predicting disease risk, understanding how diseases occur, and finding improved diagnosis and treatment strategies.

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