Key regulatory decisions for big pharma in the first half of 2013

The first half of 2013 should be notable for being the period when many of the industry’s most talked about products find out about their fate, as they run the gauntlet of regulatory approval.

Among the big events that could move the needle on companies’ share prices are Biogen Idec’s eagerly awaited multiple sclerosis treatment BG-12, which also claims the title of one of the most valuable drugs in development (Biogen and Gilead top tables with most valuable R&D assets, August 08, 2012). This will be joined by Novo Nordisk’s long-acting insulin drugs Tresiba and Ryzodeg as well as the oral blood-thinner Eliquis, which despite its late arrival to the stroke prevention world could upset the current incumbents.

Catalysts galore

While others may have one or two drugs trying to gain marketing approval, the first half of 2013 is set to be a big one for Sanofi, which has three products teed up for approval in Europe: Lyxumia in diabetes, Lemtrada in multiple sclerosis and Zaltrap in colorectal cancer.

Getting Lyxumia approved will, however, only be a stepping stone for Sanofi, given how far it is behind the market-leading GLP-1 drug Victoza. But it will be vital to moving on the proposed combination of Lyxumia, which was originated by Zealand Pharma, and Sanofi’s insulin Lantus (Event – Lyxumia decision key to Sanofi’s diabetes defence,October 11, 2012).

The other two drugs have not been without their problems.

Getting Zaltrap approved in Europe might go some way to make up for the blow the drug has suffered in the US. Last month, Sanofi was forced to halve the price of the drug following complaints from doctors at a New York hospital (Zaltrap in Europe should mean a payday for Regeneron – but pricing is problematic November 19, 2012).

Then there is the troubled Lemtrada, which has been largely eclipsed by super star BG-12 in the multiple sclerosis space. The drug suffered a refusal to file at the hands of the FDA in August and will be hoping for a slightly easier ride in Europe, as will Sanofi, which needs new products to help it address its large and painful patent cliff woes.

Waiting, waiting, waiting

It had been hoped that BG-12 might be approved in the US by the end of the year, but in October the FDA threw a large bucket of cold water over that when it extended the review period of the drug by three months to March 28. It was some small relief for eager investors that the agency did not request any additional trials and instead sought more time to review the application.

While this could say a lot about the sheer amount of data Biogen will have filed with the drug, many do not believe that there is any doubt about BG-12’s chances of approval and its rapid move into front-line multiple sclerosis treatment.

This faith in BG-12 is reflected in consensus forecasts for 2018 sales, which according to EvaluatePharma are due to hit $3.82bn. With an NPV of $11.6bn the product is second only in value to Gilead’s still risky hepatitis C candidate GS-7977.

Time could also be running out for the drug to get European approval before the officials in Brussels pack up for the Christmas holidays, but it could be a nice way for Biogen to end the year if BG-12 does squeak in.


Novo Nordisk could also get a nice holiday season surprise if the EU approves its insulin franchise of Tresiba and Ryzodeg. Tresiba is the intended brand name for the group’s long-acting, once-daily insulin, formerly known as degludec, while Ryzodeg is the intended brand name for a combination of decludec and insulin aspart Novolog.

But as previously pointed out by EP Vantage, approval could prove to be the easy part for Tresiba and Ryzodeg. The drugs are facing competition from long-acting biosimilars, which could make the new drug look expensive and complicate take-up.

US approval was expected in the first quarter, but has started to look more doubtful despite approval in Japan and the positive opinion in Europe thanks to concerns from an FDA advisory committee over excess risk of cardiovascular events in patients taking the long-acting drug over comparator products.

US approval of the oral blood-thinner Eliquis in stroke prevention is expected in March. A previous slip up with a complete response letter only concerned “data management and verification” and no new studies were needed (Eliquis FDA setback turns up the heat on Pfizer and Bristol-Myers Squibb, June 25, 2012).

With European approval in the bag approval in the US now looks likely, and the indication unlocks a key revenue stream for Bristol-Myers Squibb and Pfizer (EU backing for Eliquis in stroke prevention bodes well for US decision, November 21, 2012). According to EvaluatePharma sales for this indication are forecasted to reach $3.8bn by 2018.

Celgene’s Actimid (pomalidomide) is expecting US approval early next year, filed in the third line setting for multiple myeloma, the drug could compete with Onyx Pharmaceuticals’ Kyprolis. EvaluatePharma sales forecasts $709m by 2018 and as the company’s second largest growth driver it could help bolster its Revlimid franchise (Event – Celgene looks to poma to pick up some Revlimid slack, November 20, 2012).

Smart bomb

One group, however, that should be welcoming the start of 2013 is Roche. The Swiss company has had a good year with the US approval of Perjeta in June and is hoping to seal that success with the double of getting Trastuzumab-DM1 rubber-stamped in the US early next year (Roche scores big breast cancer win as more pivotal data approach, June 11, 2012).

With little treatment options for women and T-DM1’s sterling record so far in extending both progression-free and overall survival there should be little worry that it will reach the market by its February 28 PDUFA date.

Alongside providing more treatment options for the very sick women with metastatic Her2-positive breast cancer, approval should put antibody conjugates firmly on the treatment map.

The table below is drawn from EvaluatePharma’s Calendar of Events, and highlights some of the biggest regulatory decisions facing big pharma.

Regulatory decisions
Product Company Pharmacological class Event Indication Date Product NPV ($bn) NPV as % of Market Cap
BG-12 Biogen Idec Nuclear factor erythroid 2-related factor  pathway activator US approval MS Mar 28 11.6 32%
Tresiba Novo Nordisk Insulin US approval Diabetes Q1 6.6 9%
Ryzodeg Novo Nordisk Insulin US approval Diabetes Q1 3.4 5%
Eliquis Bristol-Myers Squibb Factor Xa inhibitor US approval Stroke prevention in atrial fibrillation Mar 15 5.4 10%
Trastuzumab-DM1 Roche Anti-HER2 (ErbB-2) MAb-DM1 maytansinoid conjugate US approval Breast cancer Feb 26 5.4 3%
Eliquis Pfizer Factor Xa inhibitor US approval Stroke prevention in atrial fibrillation Mar 15 3.5 2%
Actimid (CC-4047) Celgene Immunomodulator US approval Multiple myeloma Feb 8 2.6 8%
Canagliflozin Johnson & Johnson Sodium-glucose cotransporter-2 inhibitor EU approval, Ad Com, US approval Diabetes Q1, Jan 10, Mar 29 1.6 1%
Breo/ Relvar GlaxoSmithKline Beta 2 adrenoreceptor agonist & corticosteroid EU (COPD, asthma), Ad Com (COPD), US approval (COPD COPD, asthma Q1, Mar 7, May 12 1.4 1%
GSK2118436 GlaxoSmithKline B-Raf kinase inhibitor EU and US approval Melanoma Mar, Jun 3 1.2 1%
Lemtrada Bayer Anti-CD52 MAb EU approval MS Q2 0.8 1%
Kynamro Sanofi Apolipoprotein B-100 antisense US approval Homozygous familial hypercholesterolemia Jan 29 0.7 1%
Bedaquiline Johnson & Johnson Diarylquinoline EU approval Tuberculosis Mar 0.6 0%
Lyxumia Sanofi Glucagon-like peptide 1 agonist EU approval Diabetes Q1 0.6 0%
Lemtrada Sanofi Anti-CD52 MAb EU approval MS Q2 0.6 0%
Zaltrap Sanofi VEGFr kinase inhibitor EU approval Colorectal cancer Q1 0.5 0%
GSK1120212 GlaxoSmithKline MEK inhibitor EU and US approval Melanoma Mar, Jun 3 0.4 0%
Latuda Takeda 5-HT2A, 5-HT7 (serotonin) & dopamine D2 antagonist EU approval Schizophrenia May 0.3 1%

To contact the writer of this story email Lisa Urquhart in London at [email protected] or follow @LisaEPVantage on Twitter

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