FirstWord Lists – FDA drug approvals in 2013: Cause for industry optimism?

Set against the elongated development timelines to which pharmaceutical manufacturers operate, a period of just 12 months provides only limited visibility as to how the industry is evolving. Nevertheless, assessment of those innovative new drugs approved by the FDA during 2013 (listed in full below) does provide some insight into key sector themes.

From a numbers perspective, 2013 failed to live up to the FDA's haul of 39 new molecular entity (NME) approvals the previous year. The 27 approvals secured in 2013 is much closer to the recent historical performance of the FDA and reinforces the notion that 2012's number should be viewed as an outlier rather than any precursor to a significant increase in new drug productivity.

Rightly or wrongly, the number of innovative drugs approved by the FDA each year is also frequently cited as a barometer of the industry’s relationship with the administration. The reality is, however, that the FDA can only approve those products presented to it.

Thus, despite a number of high-profile rejections over the course of 2013 (such as Novo Nordisk’s Tresiba and Sanofi’s Lemtrada) one important theme was closer collaboration between the FDA and industry, encapsulated by introduction of the administration's Breakthrough Therapy designation.

Roche's Gazyva became the first drug benefiting from this status to gain approval, although it was the regulatory nod for Johnson & Johnson and Pharmacyclics' Imbruvica that showcased more impressively how the FDA is looking to speed up access to new drugs, particularly in oncology.

The key message, argues David Grainger – CEO at the drug development consultancy TCP Innovations – is that despite there being a third fewer innovative drugs approved in 2013, the combined five-year (post-launch) sales forecast for this crop of new drugs is potentially 10-fold that for the 'class of 2012,' should seven potential blockbuster candidates (Sovaldi, Tecfidera, Invokana, Imbruvica, Gazyva, Kadcyla and Breo Ellipta) deliver.

A major caveat is that this commercial success will benefit a small number of players. Furthermore, the two products approved in 2013 that are expected to generate the highest peak annual revenues (Gilead Sciences' Sovaldi and Biogen Idec's Tecfidera) were developed and launched by large cap biotech companies, rather than Big Pharma. Both products could generate peak sales in excess of $5 billion - indeed some analysts are suggesting that Gilead’s hepatitis C therapy could generate revenues double this benchmark. See 2013 In Review: Big biotech delivers the industry's key blockbuster franchises.

Grainger suggests therefore that although the 'class of 2013' offers means of encouragement, an underlying message that "industry R&D practices need to change remains as strong as ever." Furthermore, Grainger notes that with 46 percent of new approvals representing new mechanisms of action, the industry continues to deliver "incremental innovation."

Around a third of innovative approvals in 2013 were for oncology indications, with Roche a primary beneficiary; approval and launch of Kadcyla and Gazyva being viewed as key landmarks as the company seeks to enhance its portfolio of existing cancer therapeutics and raise the bar-to-entry for biosimilar developers (see 2013 In Review – Roche's biosimilar defence strategy gains momentum). Regulatory clearance for Imbruvica also showcased the emergence of new oral therapies in the haematological cancer space, a trend that looks poised to continue in 2014 and beyond.

Another notable trend at the therapeutic level was approval of two new respiratory medicines from the market leading player GlaxoSmithKline – Breo Ellipta and Anoro Ellipta. Commercial expectations remain high for both compounds and the next few quarters will provide greater clarity as to whether anticipated sales can be met.

At a company level, GlaxoSmithKline secured more innovative drug approvals than any other player in 2013, while Johnson & Johnson and Bayer continued to deliver new drugs to the market, thereby retaining momentum that has been developed over the past years - see FirstWord Lists: Johnson & Johnson tops Big Pharma's 2013 freshness index.

Writing for Forbes a year ago, pharma R&D expert Bernard Munos noted that 2012 had seen a dearth of innovative product approvals from the companies that needed them the most: Abbott (now AbbVie), AstraZeneca and Eli Lilly. This trend continued in 2013, although AbbVie and Eli Lilly are both expected to be more productive over the next 12 months. AstraZeneca's 'recovery' in terms of new product launches remains a longer-term narrative.

Despite the biggest approvals of 2013 emanating from the biotech sector, around 50 percent of innovative new approvals were licensed to or developed by Big Pharma companies (versus 35 percent in 2012, and an average of between 25 percent and 40 percent over the period 2004-12, according to Munos).

However, in a recent article Munos notes that only 8 of the 27 approvals in 2013 were discovered by the companies that registered them and among those drugs registered by Big Pharma players, only 3 of 14 drugs (or 21 percent) were discovered internally. A statistic that continues to focus scrutiny on how Big Pharma spends its titan R&D budgets.

FDA NME approvals 2013 (most recent first)

Anoro Ellipta (GlaxoSmithKline – chronic obstructive pulmonary disease)
Sovaldi (Gilead Sciences – hepatitis C)
Olysio (Johnson & Johnson – hepatitis C)
Luzu (Valeant Pharmaceuticals – fungal infections)
Imbruvica (Johnson & Johnson/Pharmacyclics – mantle cell lymphoma)
Aptiom (Sunovion – epilepsy)
Gazyva (Roche – chronic lymphocytic leukaemia)
Vizamyl (GE Healthcare – dementia/Alzheimer’s disease imaging)
Opsumit (Actelion – pulmonary arterial hypertension)
Adempas (Bayer – pulmonary hypertension)
Duavee (Pfizer – moderate-to-severe vasomotor symptoms)
Brintellix (Lundbeck – depression)
Tivicay (GlaxoSmithKline – HIV)
Gilotrif (Boehringer Ingelheim – NSCLC)
Mekinist (GlaxoSmithKline – melanoma)
Tafinlar (GlaxoSmithKline – melanoma)
Xofigo (Bayer – prostate cancer)
Breo Ellipta (GlaxoSmithKline – COPD)
Invokana (Johnson & Johnson – diabetes)
Tecfidera (Biogen Idec – multiple sclerosis)
Dotarem (Guerbet – MRI; brain and spine)
Lymphoseek (Navidea Biopharma – breast cancer/melanoma diagnostic)
Osphena (Shionogi – moderate to severe dyspareunia)
Kadcyla (Roche – HER2+ breast cancer)
Pomalyst (Celgene – multiple myeloma)
Kynamro (Genzyme - homozygous familial hypercholesterolaemia)
Nesina (Takeda – type 2 diabetes)

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