Novel orphan drug boom in 2015, predict experts

19-Mar-2015 - Last updated on 19-Mar-2015 at 14:45 GMT

Approvals of orphan drugs will continue to rise in 2015, says Frost & Sullivan

The pharma industry reached a high water mark in 2014 with a record number of new molecular entities approved by the US Food and Drug Administration, especially orphan drugs.

The US market for orphan therapies grew 83% compared to 2013 levels, mostly because of large unmet needs, fewer competitors and the potential for high returns, according to Jennifer Lazar, global program director for life sciences at Frost & Sullivan.

“Orphan diseases are ones that face significant unmet needs in that patients have limited to no treatment options. While the patient pool is smaller for orphan diseases than higher prevalent diseases, there is potentially higher return on investment as the pharma/biotech company can dominate that particular disease and not face the issues of price wars,” explained Lazar.

Gilead’s Zydelig

The significant increase in the approval for drugs that had a first-in-class designation can be traced back to R&D labs. “We are seeing significant advancements in areas such as next generation sequencing, understanding the genome, identifying novel biomarkers, and focusing on targeted therapy,” said Lazar. “Gilead's Zydelig is one example of a first-in-class treatment approved in 2014.”

This is the first PI3k inhibitor, which is being used to treat chronic lymphocytic leukemia (CLL), relapsed follicular B-cell non-Hodgkin lymphoma (FL), and relapsed small lymphocytic lymphoma (SLL).

Asked about the record number of new molecular entities, Lazar points to significant advances in science as it relates to identifying biomarkers and developing more targeted therapeutics. “There has been a lot more emphasis on orphan diseases and breakthrough medicines which is resulting in faster development and approval timelines, and similar to 2013, we are seeing a lot of efficiencies with the FDA and more drugs being granted expedited reviews.”

Noteworthy NMEs [new molecular entities] include BMS' Opdivo (PD-1 inhibitor) for the treatment of unresectable or metastatic melanoma and AbbVie's Viekira Pak for hepatitis C. Opdivo is the first and only PD-1 [programmed death receptor-1] therapy to demonstrate overall survival in previously treated metastatic squamous NSCLC. It is expected there is a $3bn market opportunity for the PD-1 class in melanoma, according to Frost & Sullivan’s expert.

She also noted a lack of clear regulatory pathway for biosimilars in the US market in 2014, with no biosimilars approved, but Lazar predicts this will change in 2015, ushered in by the FDA’s approval of Sandoz's Zarxio (biosimilar to Amgen Inc.’s Neupogen).

“Now that the first biosimilar has been approved, I would expect many more to follow and with a clearer set of guidelines.”

Lazar’s report is titled “Regulatory Trends in the US Pharma/Biotech Industry: Efficiency of the FDA Review Process Expedites Access to Care”