FirstWord Lists: The drugs that will shape 2013 – Key regulatory catalysts

A significant increase in the number of FDA approvals during 2012 has caused room for industry optimism. As a result, there will be notable focus over the next 12 months on how a raft of new launches perform commercially (see FirstWord Lists: The drugs that will shape 2013 – new/recent launches).

To retain momentum, however, leading players must also ensure a succession of new approvals in 2013. Nevertheless, approvals alone are no guarantee of a net positive return on investment for the pharmaceutical industry (see Spotlight On: FDA Approvals in 2012 – Winners and Losers).

Thus focus will remain sharpened on those regulatory catalysts – be they PDUFA dates, European approvals or AdCom meetings – that will shape the route to market for potential blockbuster therapies and continue to build growth sentiment for the industry-at-large.

Those regulatory catalysts listed below are not designed to represent an exhaustive list (and not included are a number of anticipated approvals in Europe for key products recently approved by the FDA; such as Pfizer's Xeljanz). Notably, many of the industry's key regulatory catalysts are expected to occur early in 2013, which could further emphasise drug launches (rather than approvals or Phase III read-outs) as the most important theme of the year.

BG-12 (dimethyl fumarate) – Biogen Idec (multiple sclerosis) | PDUFA date – March 28

Based on current consensus forecasts, Biogen Idec's BG-12 – a third-to-market oral treatment for multiple sclerosis – is expected to be the industry's most lucrative approval of 2013. Current analyst consensus forecasts compiled by FirstWord indicate global sales of $3.3 billion by 2017 (see FirstWord Lists: Pharma's largest pipeline opportunities).

Although both Novartis' Gilenya and Sanofi's Aubagio have launched in the oral setting, BG-12 is viewed as the superior therapy; with commercial expectations heightened by the fact that Gilenya is nearing global sales of $1 billion some 18 months after launch (see FirstWord Lists: Multiple sclerosis winners and losers in 2016). International Strategy and Investment analyst Mark Schoenebaum recently noted that Biogen Idec management has sought to play down the very highest peak sales forecasts for BG-12, but confidence in the product remains undiminished with a full sales force already in place. Teva's recent move to delay approval is a further sign that competitors are wary as to what percentage of market share they are likely to lose to BG-12 (see Spotlight On: Teva's attempts to block approval of new multiple sclerosis therapies).

Breo/Relvar (fluticasone furoate/vilanterol) – GlaxoSmithKline (asthma/COPD) | AdCom date – March 7; PDUFA date – May 13

As recently noted in a number of quarters, 2013 stands to be a critical year for GlaxoSmithKline; both in terms of regulatory submissions and potential approvals (see ViewPoints: GlaxoSmithKline looks to deliver on pipeline aspirations (again)).

For many investors, the most significant catalyst will be the AdCom meeting in March that will focus on Breo/Relvar for the potential once-daily treatment of chronic obstructive pulmonary disease (COPD) and its subsequent PDUFA date towards mid-year. At the recent JP Morgan Healthcare conference, GlaxoSmithKline management suggested that topics for discussion at the forthcoming AdCom are likely to be once versus twice-daily dosing (critical in terms of switching patients from the older Seretide/Advair franchise and defending market leadership from potential rival once-daily treatments) and a pneumonia signal seen in the Phase III programme. Should these obstacles be navigated and approval secured, focus (particularly in Europe) will switch to the company's ability to switch patients and drive uptake. Relvar is currently undergoing a 'real world' study (SALDORD) designed to address this post-launch challenge and gain support from payors (see ViewPoints: Will European austerity measures prompt structural changes at GlaxoSmithKline?).

T-DM1 (trastuzumab) – Roche (second-line HER2-positive breast cancer) | PDUFA date – February 26

Having secured approval for Perjeta in 2012, regulatory clearance for T-DM1 – Roche's conjugated antibody – represents the second element of a double pronged strategy to both defend and enhance the company's dominant position in HER2-positive breast cancer. Analysts are highly confident of approval given the strong data that T-DM1 has demonstrated, and regulatory clearance will mark a notable landmark in the space for ADC products and validate this technology. Trastuzumab has received priority review as a third-line therapy for HER2-positive breast cancer, but data set to be published by mid-2013 in combination with Perjeta (versus Herceptin and taxane) could allow Roche to replace Herceptin completely, and effectively substitute one of its leading franchise with a new one, benefiting from greater patent exclusivity and higher pricing economics (see ViewPoints: Roche continues to build case for breast cancer dominance).

Tresiba (insulin degludec) – Novo Nordisk | PDUFA date – not specified

Anticipated FDA approval of Novo Nordisk's long-acting insulin product Tresiba (following a positive AdCom assessment in late 2012) will have a number of implications. Novo Nordisk's position as one of the strongest industry players – a status built on its specialty focus in the high-growth diabetes segment – would be further enhanced, as would the competitive threat to Sanofi's Lantus; the current market leader in the long-acting insulin space. Lantus now generates a considerable proportion of Sanofi's revenues and estimated profits, and as the French company looks to reduce its dependency on the franchise via advancement of its own pipeline, approval of Tresiba in the key US market will represent a notable challenge. Novo Nordisk is seeking to differentiate its own product from Lantus, primarily via a superior hyperglycaemic side-effect profile; analysts are increasingly confident of approval (although the company may be required to undertake a pre-approval CV outcomes study); however, the details of commercial uptake may be in the labelling. See ViewPoints: A messy marketing battle awaits in diabetes market and Spotlight On: Defence of key brands remains paramount at Sanofi and Novartis.

Pomalidomide – Celgene (multiple myeloma) | PDUFA date – February 10

On the back of the annual JP Morgan healthcare conference, Celgene was the best performing biotech company last week, with robust share price performance driven by higher-than-anticipated long-term revenue and earnings guidance for 2017. The biotech sector enjoyed a robust 2012, but there is creeping sentiment among some analysts that valuations are becoming 'full' if yet overstretched. Others suggest that the recent suggestion that biotech has gained ground on its Big Pharma rivals in terms of M&A clout may prove prophetic; could a pipeline gap over the next few years drive a biotech purchasing spree? Thus, beyond the biotech poster-story stocks of Biogen Idec and Gilead Sciences – expected to deliver the two biggest new products to the market by 2017 – Celgene's performance (and if it can deliver on its long-term guidance) will be key to sector sentiment. Last week management labelled pomalidomide as the company's "next blockbuster," with Onyx Pharmaceuticals' Kyprolis providing a recent benchmark for an anticipated strong launch (see ViewPoints: Q3 results show more cancer blockbusters in the making.

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