Key regulatory events for the next six months in Q1 and Q2 2012

The first half of the year is shaping up to hold a number of key regulatory decision for the industry's smaller drug makers, with make or break decisions due for Vivus' obesity drug Qnexa, Map Therapeutics' migraine treatment Levadex and Protalix's shot at breaking into the Gaucher market, Uplyso, with a host of others (see table).

Of course, these decisions are unlikely to all yield good news, as Allos Therapeutics found today, when European regulators refused to grant approval for Folotyn. Filed to treat peripheral T-cell lymphoma, the CHMP ruled the single arm registration study, which did not measure overall survival, failed to establish the drug's benefit. Many were expecting approval after the FDA granted approval in the same setting in 2009; Allos shares were trading 13% lower at $1.45 in early trade today. The companies below will be hoping for a more positive outcome.

Disease modifying

Vertex will be counting down the days to April when it is due to hear from US and EU regulators on Kalydeco, the company’s cystic fibrosis drug. The drug targets the defective protein that causes cystic fibrosis and would be the first treatment to treat the underlying cause of the disease. Currently the treatment is for only patients who carry a specific gene mutation. However the company is planning additional studies in children as young as two and patients with mutations who have not been evaluated in the previous trials (Vertex's cystic fibrosis candidate exceeds hopes, February 24, 2011)

As Qnexa is its biggest growth driver, Vivus awaits a number of regulatory milestones in the first half of the year for the obesity drug. With confirmation back in December that a component of the product is associated with a higher rate of oral clefts in the children exposed to the drug in the womb, it is unknown whether regulators will be convinced that the benefits outweigh the risks.

Then April 29 sees the PDUFA for Vivus' erectile dysfunction drug Avanafil. Aside from the regulatory hurdles facing the product, its commercial outlook will depend a lot on whether generics manage to overturn a surprise ruling last year that could protect Pfizer's Viagra out until 2019 (Pfizer wins Viagra US patent spat, August 15, 2011)

Meanwhile US regulators will decide on Map Pharmaceuticals' inhaled migraine drug Levadex at the end of March. The company announced at the start of last year a collaboration with Allergan to sell the drug in the US. If Levadex reaches forecasted sales of $331m in 2016 it could be the biggest-selling branded anti-migraine therapy on the market, forecast data from EvaluatePharma show. However, with regulators proving tough on inhaled products outside the respiratory space and the drug attempting to crack into the crowded and highly genericised migraine market, those numbers could prove optimistic (Regulatory worries dampen enthusiasm for Map's Levadex deal, February 2, 2011)

It is crunch time for Ironwood this year, with its irritable bowel syndrome treatment linaclotide up for approval in the both the US and Europe. Partners Forest Laboratories and Almirall will also be keenly awaiting the news, along with the regulators' verdict on their COPD product Elkira.

Drawn from EvaluatePharma's Calendar of Events, the table below highlights some of the biggest regulatory decisions facing the industry, outside of big pharma. An analysis of clinical trial read outs and patent decisions will follow.

Regulatory decisions        
Product Pharmacological Class Company Event Date Product  NPV ($m) NPV as % of Market Cap Market Cap ($m)
Kalydeco CFTR potentiator Vertex Pharmaceuticals PDUFA/EMA decision date Apr 18/Q2 2,536 +33% 7,711
Qnexa Adrenoreceptor agonist & anti-convulsant VIVUS EMA decision date/FDA AdCom/PDUFA Q1/Feb 22/Apr 17 1,342 +122% 1,099
Levadex 5-HT1B (serotonin) & 5-HT1D (serotonin) agonist MAP Pharmaceuticals PDUFA Mar 26 1,126 +271% 415
Linaclotide Guanylate cyclase type-C receptor agonist Forest Labs PDUFA Jun 8 957 11% 8,610
Linaclotide Guanylate cyclase type-C receptor agonist Ironwood Pharmaceuticals EMA decision date/PDUFA Q2/Jun 8 899 +49% 1,816
Linaclotide Guanylate cyclase type-C receptor agonist Almirall EMA decision Q2 109 9% 1,200
Gattex Glucagon-like peptide 2 (GLP-2) agonist NPS Pharmaceuticals EMA decision date/PDUFA Mar 1/Q2 821 +140% 588
RG3616 (GDC-0449) Hedgehog pathway/smoothened (SMO) inhibitor Curis PDUFA Mar 8 814 +222% 366
Ridaforolimus Rapamycin analogue (mTOR inhibitor) ARIAD Pharmaceuticals PDUFA June 5 615 +27% 2238
Peginesatide Erythropoietin stimulator Affymax PDUFA Mar 27 483 +190% 255
Eklira Long-acting muscarinic antagonist (LAMA) Forest Labs PDUFA  Apr 30 308 +4% 8,610
Eklira Long-acting muscarinic antagonist (LAMA) Almirall EMA decision date Q2 350 +29% 1,220
Dacogen DNA methyltransferase (DNMT) inhibitor Astex Pharmaceuticals FDA AdCom/PDUFA/EMA decision date Feb 9/Q1/Q2 322 +161% 200
Northera Norepinephrine precursor Chelsea Therapeutics FDA AdCom/PDUFA Feb 23/Q1 264 +79% 333
Bydureon Glucagon-like peptide 1 (GLP-1) agonist Amylin Pharmaceuticals PDUFA Q1 260 +15% 1,697
Avanafil Phosphodiesterase V (PDE5) inhibitor VIVUS PDUFA Q2 256 +23% 1,099
Uplyso Glucocerebrosidase Protalix BioTherapeutics PDUFA May 1 246 +49% 502
Korlym Selective glucocorticoid receptor (GR-II) antagonist Corcept Therapeutics PDUFA Feb 17 205 +73% 281
Budesonide MMX Corticosteroid Cosmo Pharmaceuticals EMA decision date H1 162 +56% 291
Crinone Progestogen agonist Columbia Laboratories FDA AdCom/PDUFA Jan 20/Q1 125 +91% 138
Pixuvri Anthracycline Cell Therapeutics EMA decision date/FDA AdCom/PDUFA Q1/Feb 9/Apr 24 0   228

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