Beyond borders: biotechnology industry report 2013

Products and pipeline

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In 2011, the number of FDA product approvals increased significantly for the first time in seven years. In 2012, the number of FDA approvals increased even further, to levels not seen since 1997.

To put that in context, the last time the FDA approved this many products, the president of the United States was Bill Clinton and the human genome had not yet been sequenced. Similar strength was apparent in Europe, where a number of significant products were approved by regulators.

We are encouraged not just by the number of approvals but by the nature of products being approved. Many of the year’s approvals were not me-too offerings but first-in-class treatments that seek to address genuine unmet needs. Significant numbers of new products are for orphan indications and/or based on personalized medicine approaches.

This is encouraging news for investors concerned about regulatory risk and the time and expense associated with developing new products. It is potentially good news for pharma companies seeking to replenish their pipelines. Ultimately, of course, it is positive news for the many patients who need breakthrough treatments for increasingly urgent health challenges.

United States

FDA product approvals, 1996–2012

FDA product approvals, 1996–2012

The number of product approvals — new molecular entities (NMEs) and biologic license applications (BLAs) — by the US Food and Drug Administration (FDA) soared for the second consecutive year. In 2012, the FDA approved 39 products: 33 NMEs and 6 BLAs. This was the highest number of FDA approvals since 1997.

Perhaps as a reaction to the criticism the agency has come under from industry and patient groups seeking access to medicines, the FDA explicitly highlighted not just the large number of approvals but also the speed with which they had been approved and the fact that many of the products were innovative medicines that addressed genuine unmet needs (51% were first-in-class medications and 33% were orphan drugs).

Twelve of the products approved were in oncology, while gastrointestinal and respiratory indications saw four approvals each.


European-specific biotech findings

Like the US, Europe saw a number of orphan drug approvals in 2012. Glybera became the first gene therapy drug approved in the Western world when Netherlands-based UniQure secured approval for the product in October. Gene therapy, an area that was considered very promising in biotech’s early years, was bedeviled for years with R&D setbacks and safety concerns. The approval of Glybera, a therapy for a rare disease which leaves people unable to properly digest fats, is therefore a notable achievement.

To view our complete findings on this topic, download the full report

FDA product approvals, 1996–2012

FDA product approvals, 1996–2012

Source: EY, FDA.
US product approvals are based on CDER approvals only.


Selected orphan drug approvals by the EMA, 2012 Company Brand

Company Brand name Generic name Indication Month
Nova Laboratories Xaluprine 6-mercaptopurine monohydrate Acute lymphoblastic leukemia March
Pharmaxis Bronchitol Mannitol Cystic fibrosis April
Novartis Signifor Pasereotide diaspartate Cushing's disease April
Vertex Pharmaceuticals Kalydeco Ivacaftor Cystic fibrosis July
Novartis Jakavi Ruxolitinib Disease-related splenomegaly or symptoms of primary myelofibrosis, post-polycythaemia-vera myelofibrosis or post-essential-thrombocythaemia August
Takeda (Nycomed) Revestive Teduglutide Short-bowel syndrome August
Johnson & Johnson (Janssen-Cilag) Dacogen Decitabine Acute myeloid leukemia September
Takeda (Millenium) Adcetris Brentuximab vedotin Hodgkin lymphoma October
uniQure Glybera Alipogene tiparvovec Familial lipoprotein lipase deficiency October
Teva (Ivax) NexoBrid Concentrate of proteolytic enzymes enriched in bromelain Thermal burns December

Source: EY, EMA and company websites.