Midcap players ask Europe to bring it on in 2013

If the European regulator has sometimes given the impression of being a mere rubber-stamper of its US opposite number’s decisions, this year it certainly showed that it has a mind of its own.

And in the first half of 2013 it is the EU that will be the battleground for midcap players’ key regulatory decisions; many will concern products already given the US green light, such as Medivation’s Xtandi, Arena Pharmaceuticals’ Belviq and Ariad Pharmaceuticals’ ponatinib (see table below). Midcap pharma and biotech will thus watch eagerly for continued signs of independence of thought.

Among the drugs with the highest NPV as calculated by EvaluatePharma, it is perhaps the EU decision facing the obesity pill Belviq that should cause most concern. The read-across comes from Vivus’s competitor Qsymia, which did at long last secure US approval last year, but then fell over at the CHMP.

Tough stance

As far as obesity goes Europe is clearly taking a tough stance, likely relating to risk/benefit considerations. Although normally EU sales represent no more than a significant minority of revenues, the territory forms a major part of Arena’s strategy – hence the importance of the EU verdict on Belviq.

Meanwhile, Xtandi and ponatinib – highly promising cancer drugs – likely have little to fear, although investors will watch eagerly for any additional fallout from the safety issues that recently led the FDA to add a warning to ponatinib’s label (Ariad approval catches bio run-up traders by surprise, December 17, 2012).

Still, the EMA has shown that it can go boldly where the FDA fears to tread: for instance, although Pfizer’s blood thinner Eliquis was hit with a US complete response letter citing data management and verification issues the EU regulator had no qualms about giving it a clean bill of health. This stance could embolden Ariad.

Also awaiting the European green light at some point by mid-2013 is Dendreon’s hugely hyped prostate cancer autologous immunotherapy, Provenge.

US triggers too

Meanwhile, of the most important midcap US catalysts in the next six months the biggest is surely the FDA decision on Theravance’s LABA/steroid combination Breo. This will face a US panel for the COPD indication before a US decision due by March 12. Breo, formerly known as Relovair, carries an NPV of $4.1bn, according to EvaluatePharma data, and could become Theravance’s first approved drug and transform the company.

Analysts still see considerable risk, given the heterogeneity of phase III data and LABA dose concerns; it is uncertain when a US filing for asthma – a problematic indication – might later be made. Breo also faces an EU decision, under the name Relvar, for use both in asthma and COPD.

A broader consideration is that Theravance’s partner GlaxoSmithKline will likely manage the launch process carefully given the current dominance in both asthma and COPD of its own blockbuster, Advair. The companies’ follow-up COPD combination of umeclidinium and vilanterol has just been filed in the US.

Also in the US, ImmunoGen is looking forward to approval of its antibody-drug conjugate Trastuzumab-DM1, following its extension of both progression-free and overall survival in metastatic Her2-positive breast cancer. The product’s approval is a major catalyst for ImmunoGen’s partner Roche, too (Key regulatory decision for big pharma in the first half of 2013, December 20, 2012).

And rare diseases continue to feature on investors’ radars. Here, a duo of small companies – Isis Pharmaceuticals and Aegerion Pharmaceuticals – await regulatory verdicts in the tiny indication of hereditary familial hypercholesterolaemia. The latter’s lomitapide has just secured US approval – hardly surprising given its strong advisory panel endorsement – and awaits an EU ruling.

For Isis’s Kynamro, however, the jury in the US is still out. Not only was its panel backing more cautious, but to add to Isis’s woes the CHMP recently issued a negative opinion on Kynamro, citing safety concerns – another clear example that a positive US outcome is no longer a reliable indicator of the regulatory verdict on the other side of the pond.

The table below is drawn from EvaluatePharma’s Calendar of Events, and highlights some of the biggest regulatory decisions facing mid and micro-cap pharma and biotech companies in the first half of 2013.

To contact the writers of this story email Jacob Plieth or Joanne Fagg in London at [email protected] or follow @JacobEPVantage and @JoEPVantage on Twitter