Precision medicine has been capturing the headlines big time in the past couple of weeks with major initiatives being created in the U.S. and the UK. Their objective is to marshal resources to be able to accelerate the discovery and delivery of targeted therapies to patients more effectively.

The personalized approach to treatment has certainly come a very long way during the past decade. It first emerged on the health care agenda as an exciting future concept and has since evolved rapidly to the present day where it has become the mantra in any discussions relating to 21st century medicine. Its influence is accelerating as witnessed by the FDA approvals of targeted medicines, whose number has grown significantly each year. This will come as no surprise given the importance of the field with its potential to improve the quality of care and treatment outcomes.

For example, it is not uncommon for new medicines under development to now have a parallel companion diagnostic or biomarker program designed to assist in speeding development times and ultimately to help identify the appropriate patients most likely to benefit from the targeted therapies. In fact, in 2014, according analysis conducted by the Washington-based Personalized Medicine Coalition, more than 20 percent of the 41 new drugs that received the green light from the FDA's Center for Drug Evaluation and Research (CDER) are personalized medicines.

In carrying out its assessment, the PMC said it undertook a detailed review of available data provided by the FDA on the approved products. In order to qualify as a personalized medicine the therapy's product label needed to refer to a specific biomarker, identified by diagnostic tools, that can play a role in treatment decision making – whether or not to treat or how to treat – for an individual patient.

With this definition in hand their analysis identified nine newly approved personalized medicines from the crop of new medicines that were approved for use. These, with the reason why each was identified by PMC, included:

Astrazeneca plc's Lynparza (olaparib), a PARP (poly ADP-ribose polymerase) inhibitor for the treatment of advanced ovarian cancer. The decision to treat with this product is affected by the BRCA biomarker status in patients.

Lynparza was approved as a monotherapy in patients with deleterious or suspected deleterious germline BRCA-mutated advanced ovarian cancer who have been treated with three or more prior lines of chemotherapy. The BRCA mutations, occurring in 15 percent of ovarian cancers, prevent homologous recombination, the DNA repair process that kicks in if PARP is inhibited. That means PARP inhibition can kill tumor cells without affecting normal cells. (See BioWorld Today, Dec. 22, 2014.)

The FDA approved Lynparza with a genetic test called BRACAnalysis CDx, a companion diagnostic that will detect the presence of mutations in the BRCA genes (gBRCAm) in blood samples from patients with ovarian cancer.

Myriad Genetics Inc., of Salt Lake City, received FDA approval for the BRCA analysis test to be used in conjunction with Lynparza.

Biomarin Pharmaceutical Inc.'s Vimizim (elosulfase alpha) for the treatment of Mucopolysaccharidosis Type IV (Morquio A syndrome). Morquio A syndrome is a rare, autosomal recessive lysosomal storage disease caused by a deficiency in N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Vimizim is intended to replace the missing GALNS enzyme whose absence leads to problems with bone development, growth and mobility. There are approximately 800 patients with Morquio A syndrome in the U.S. (See BioWorld Today, Feb. 18, 2014.)

The decision to treat with this product is affected by the type A or B biomarker status in patients.

Cyrazma (ramucirumab, Eli Lilly and Co.) for use as a single agent for the treatment of patients with advanced or metastatic gastric or gastroesophageal junction adenocarcinoma whose disease has progressed during or after prior treatment with fluoropyrimidine- or platinum-containing chemotherapy. Ramucirumab is a recombinant monoclonal antibody of the IgG1 class that binds to vascular endothelial growth factor receptor-2 (VEGFR-2) and blocks activation of the receptor. Treatment procedures are influenced by the EGFR or ALK biomarker status in patients.

Zykadia (ceritinib, Novartis) for patients with a certain type of late-stage non-small cell lung cancer (NSCLC). The drug is an anaplastic lymphoma kinase (ALK) tyrosine kinase inhibitor that blocks proteins that promote the development of cancerous cells. It is intended for patients with metastatic ALK-positive NSCLC who were previously treated with crizotinib, the only other approved ALK tyrosine kinase inhibitor. According to the FDA about 85 percent of lung cancers are NSCLC, making it the most common type of lung cancer. However, only 2 percent to 7 percent of patients with NSCLC are ALK-positive.

The decision to treat with this product is affected by the ALK biomarker status in patients.

Spectrum Pharmaceuticals Inc.'s Beleodaq (belinostat) for the treatment of peripheral T-cell lymphoma (PTCL), which comprises a diverse group of rare diseases in which lymph nodes become cancerous. Patients with the disease account for 10 percent to 15 percent of the estimated 70,800 non-Hodgkin's lymphoma cases in North America, according to the National Cancer Institute.

Treatment procedures are influenced by the UGT1A1 biomarker status in patients.

Cerdelga (eliglustat, Genzyme unit of Sanofi SA) for the long-term treatment of Gaucher disease type 1. Treatment procedures are influenced by the CYP2D6 biomarker status in patients.

Harvoni (ledipasvir and sofosbuvir, Gilead Sciences Inc.) for the treatment of chronic hepatitis C infection. The decision to treat with this product is affected by the genotype 1 biomarker status of the viral infection in patients.

Viekira Pak (ombitasvir, paritaprevir, and ritonavir; dasabuvir, Abbvie Inc.) for the treatment of chronic hepatitis C infection. The decision to treat with this product is affected by the genotype 1 biomarker status of the viral infection in patients.

Blincyto (blinatumomab, Amgen Inc.) for the treatment of B-cell precursor acute lymphoblastic leukemia (ALL). The decision to treat with this product is affected by the Philadelphia chromosome biomarker status in patients.

This number of personalized medicines could accelerate significantly in the coming years if the bold new precision medicine initiatives exert their influence as planned. In addition to the president's recently announced Precision Medicine Initiative, the UK rolled out its own national initiative in precision medicine with a war chest of £200 million (US$304.7 million). (See BioWorld Today, Feb. 12, 2015.)

The initiative plans to promote the development of tools, processes and systems that are needed to identify the right therapy, for the right patient at the right time and right dose.

"It is clear that personalized medicine is increasingly becoming an integral part of clinical care and we expect this trend to continue along with greater recognition of the value of personalized medicine by payers and providers," said Daryl Pritchard, vice president, science policy, PMC.

Editor's note: See next week's issue which looks at the planned programs under the Precision Medicine Initiative.