Citeline Pharma R&D Annual Review 2015 Supplement: New Active
Citeline Pharma R&D Annual Review 2015 Supplement: New Active Substances Launched During 2014 Ian Lloyd Senior Director, Pharmaprojects/Pipeline and Data Integration Following on from our review of trends in the current pharmaceutical R&D pipeline, published in early February 2015, this supplement takes a look at the industry’s success stories of 2014 – the drugs which were launched on to the market for the first time during the year. Our survey focuses exclusively on new active substances (NASs): new chemical or biological entities where the active drug had received no prior approval for human use. As such, this list represents a subset of all the first launches which Citeline reported during 2014, excluding as it does the 51 new drug launches with reformulated or non-NAS moieties, or biosimilars. This year, our 2015 Pharma R&D Report showed the pipeline universe to be expanding at an unprecedented rate and in all dimensions. More drugs are in development than ever before, at all phases, and in all therapeutic areas. But a cynic might suggest that all this energy is being wasted unless it leads to the birth of new star products – drugs actually successfully reaching the market. This supplement thus adds this perspective, and we’ll look not only at overall numbers, but at drug types, the leading players, and crucially, which of the new stars in the pharma firmament are shining with novel properties. And in short, it looks as though 2014 was nothing short of dazzling! 2 Sixty-one NASs – we have lift off! With no fewer than 61 new active substances reaching the market for this first time in 2014, last year broke all records. Just how outstanding the last twelve months have been is visible clearly in Figure 1, which looks at the NAS launches by year since the turn of the millennium. This shows that 2014 soared past the previous record-holder, 2013, to take the crown as the best year ever, leaving that year’s total of 48 NASs for dust. As before, we’ve also highlighted how many of the total can be accounted for by vaccines, as quite often a public health crisis, such as bird ‘flu, can lead to a flurry of launches of novel but ostensibly similar new vaccine products in response. There were only six vaccines launches in 2014, and these were a relatively diverse bunch; the next surge will likely be a brace of Ebola vaccines which will hopefully come to the market in 2015 or 2016. Figure 1. Number of NAS Launches by Year, 2000–2014, with Numbers Excluding Vaccines Also Shown for 2007–2014 70 Vaccines Other NASs 60 6 50 40 8 1 30 20 1 38 39 10 0 33 35 31 28 26 31 11 1 3 11 55 35 36 37 37 2011 2012 2013 26 17 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2014 Source: Pharmaprojects ®, February 2015, Citeline So the underlying, vaccine-free picture is even stronger: 55 new molecules in 2014, compared with 35-37 in each of the four previous years. This puts this period light years ahead of any others. But will 2014’s meteoric rise also turn out to be as transient as a shooting star? Arguably, productivity at this rate must be sustained if the increases in pipeline size we’ve already seen are going to be supported by new income. But one could at a stretch apply a line of best fit along the data from 2007 onwards to indicate a sustained upward trajectory. However, a few late-stage failures this year could easily see things crash to Earth just as quickly, so it’s best not to get too hyperbolic just yet. 3 The 2014 NAS stats: novelty, breakthroughs, but mixed-fortunes for big pharma The full alphabetical list of NASs for 2014 is collected in Table 1. Included in the table are the drugs’ generic and trade names, their mechanisms of action, the companies which have launched them, the diseases they are launched for, and the country and month of first launch. We have also included an indication of whether or not they have received orphan drug status in at least one market, and whether or not they can be considered novel in terms of whether a drug with the same mechanism(s) is already on the market or not. This last metric, highlighted in the column headed ‘First in Class’ reveals that twelve of 2014’s debutant drugs are novel, indicating a good year for innovation too. There were ten in 2013, so the proportion is very slightly lower. But if anyone a year ago had offered the industry a year with 61 NASs of which almost 20% are novel, I’m pretty sure it would have said ‘”Yes, please!”. Table 1. New Active Substance Launches 2014 Generic name 4 Trade name Company Indication Mechanism of action Country of first launch Month of first launch First in Class Orphan Drug Status USA July No No albiglutide Tanzeum GlaxoSmithKline Type 2 diabetes Glucagon-like peptide 1 agonist alectinib hydrochloride Alecensa Roche Non-small cell lung cancer ALK receptor tyrosine kinase inhibitor Japan Sept No Yes allisartan isoproxil Xinlitan Allist PharmaceutiHypertension cals / Shenzhen Salubris Angiotensin II 1 antagonist China June No No anti-H5N1 avian influenza antibodies, Fab’entech Fabenflu Fab’entech Influenza virus infection prophylaxis Immunostimulant AsiaPacific March No Yes apremilast Otezla Celgene Psoriatic arthritis Phosphodiesterase 4 inhibitor / Tumour necrosis factor alpha antagonist USA April No Yes asunaprevir Sunvepra Bristol-Myers Hepatitis-C Squibb infection HCV nonstructural protein 3 inhibitor Japan Sept No No ataluren Translarna PTC Therapeutics Germany Dec Yes Yes belinostat Beleodaq TopoTarget / Peripheral T-cell Histone deacetySpectrum lase inhibitor Pharmaceuti- lymphoma cals USA Aug No Yes blinatumomab Blincyto Amgen Acute lymphocytic leukaemia CD19 antagonist USA Dec Yes Yes conbercept Compaq Sipp Chengdu Kanghong Macular degeneration, age-related, wet Vascular endothelial growth factor (VEGF) receptor antagonist China Feb No No daclatasvir Daklinza Bristol-Myers Hepatitis-C Squibb infection HCV nonstructural protein 5A Germany inhibitor Aug Yes No dalbavancin Dalvance Durata Therapeutics Cell wall synthesis inhibitor July No No Duchenne’s muscular dystrophy Acute bacterial skin and skin structure infections Ribosomal readthrough stimulant USA Generic name Trade name Company Indication Mechanism of action Country of first launch Month of first launch First in Class Orphan Drug Status dasabuvir Exviera AbbVie Hepatitis-C infection HCV nonstructual protein 5B inhibitor USA Dec No No defibrotide Defitelio Gentium Severe hepatic veno-occlusive disease Unidentified pharmacological activity Austria March No Yes delamanid Deltyba Otsuka Tuberculosis Cell wall synthesis inhibitor UK May No Yes Duramed (Teva) Adenovirus infection prophylaxis Immunostimulant USA Oct No No adenovirus Type 4 and Type 7 vaccine, live, Teva dulaglutide Trulicity Eli Lilly Type 2 diabetes Glucagon-like peptide 1 agonist USA Nov No No efinaconazole Jublia Kaken Pharmaceuti- Onychomycosis cal / Valeant Cell wall synthesis inhibitor USA July No No efraloctocog alfa Eloctate Biogen Idec Haemophilia A Factor VIII stimulant USA July No Yes eftrenonacog alfa Alprolix Biogen Idec Haemophilia B Factor IX stimulant USA May No Yes eliglustat Cerdelga Sanofi (Genzyme) Gaucher’s disease Ceramide glycosyltransferase inhibitor USA Aug Yes Yes elosulfase alfa Vimizim BioMarin Mucopolysaccharidosis IV N Acetylgalactosamine 6 sulfatase stimulant USA Feb No Yes empagliflozin Jardiance Boehringer Ingelheim / Eli Lilly Type 2 diabetes Sodium/glucose cotransporter 2 inhibitor USA Aug No No Biotest Hepatitis-B infection in neonates Immunostimulant Vietnam Feb No No Gilead Sciences Relapsed chronic lymphocytic leukaemia / Non-Hodgkin’s lymphoma PI3 kinase delta inhibitor EU/USA Oct Yes Yes GlaxoSmithKline / Kaketsuken Influenza virus infection prophylaxis Immunostimulant Japan March No Yes Influenza virus infection prophylaxis Immunostimulant India July No No Type 2 diabetes Sodium/glucose cotransporter 2 inhibitor Japan April No No Luqa Gastrointestinal Pharmaceutidisease cals Unidentified pharmacological activity China & Hong Kong June No No hepatitis-B immunoglobulin Fovepta for neonates, Biotest idelalisib Zydelig influenza vaccine, pandemic, EB66, GSK influenza vaccine, trivalent, LAIV, Serum Institute of India Serum Nasovac-S Institute of India ipragliflozin Suglat LQ-006 Astellas / Kotobuki luseogliflozin Lusefi Taisho / Novartis Type 2 diabetes Sodium/glucose cotransporter 2 inhibitor Japan May No No meningococcal B vaccine, Novartis Bexsero Novartis Meningococcal infection prophylaxis Immunostimulant EU Jan No No 5 Generic name 6 Trade name Company Indication Mechanism of action Country of first launch Month of first launch First in Class Orphan Drug Status meningococcal B vaccine, Pfizer Trumenba (suspension) Pfizer Meningococcal infection prophylaxis Immunostimulant USA Nov No No netupitant + palonosetron hydrochloride, Helsinn* Helsinn / Eisai Radio/ chemotherapyinduced nausea and vomiting Neurokinin 1 receptor antagonist USA Oct No No Idiopathic pulmoary fibrosis VEGFR-2 tyrosine kinase inhibitor / Platelet-derived growth factor receptor kinase inhibitor / Src inhibitor / Lymphocytespecific tyrosine kinase inhibitor / LYN tyrosine kinase inhibitor / FGF receptor tyrosine kinase inhibitor / Vascular endothelial growth factor (VEGF) receptor antagonist / VEGFR tyrosine kinase inhibitor USA Oct No Yes PD-1 antagonist / Anticancer immunotherapy Japan Sept Yes Yes USA Jan No Yes Akynzeo nintedanib Ofev Boehringer Ingelheim nivolumab Opdivo Ono / Bristol-Myers Melanoma Squibb obinutuzumab Gazyva Roche / Biogen Idec Chronic lymphocytic leukaemia CD20 antagonist olodaterol Striverdi Boehringer Ingelheim Chronic obstructive pulmonary disease Long-acting beta 2 adrenoceptor (LABA) agonist EU April No No ombitasvir + paritaprevir + ritonavir** Viekira pak AbbVie Hepatitis-C infection HCV non-structural protein 3 inhibitor / HCV non-structural protein 5A inhibitor USA Dec No No oritavancin Orbactiv Eli Lilly / Targanta Acute bacterial skin and skin structure infections Cell wall synthesis inhibitor USA Nov No No pembrolizumab Keytruda Merck & Co Melanoma PD-1 antagonist / Anticancer immunotherapy USA Oct No Yes rabies vaccine, Zydus Cadila Vaxirab N Zydus Cadila Rabies infection prophylaxis Immunostimulant India Jan No No ramucirumab Cyramza Eli Lilly (ImClone) / Dyax Stomach and oesophageal cancer Vascular endothelial growth factor receptor 2 antagonist USA July Yes Yes ripasudil hydrochloride hydrate Glanatec D. Western Therapeutics / Kowa Glaucoma Rho-associated kinase inhibitor Japan Dec No No serelaxin Reasanz Novartis Heart failure Relaxin agonist Russia April Yes No siltuximab Sylvant Johnson & Johnson Castleman’s disease Interleukin 6 antagonist USA May No Yes Generic name Trade name Company Indication Mechanism of action Country of first launch Month of first launch First in Class Orphan Drug Status UK Oct No No simoctocog alfa Nuwiq Octapharma Haemophilia A Factor VIII stimulant sofosbuvir + ledipasvir*** Harvoni Gilead Sciences Hepatitis-C infection HCV nonstructural protein 5A inhibitor / HCV nonstructual protein 5B inhibitor USA Oct No No sucroferric oxyhydroxide Velphoro Galenica, Switzerland / Fresenius Hyperphosphataemia Phosphate antagonist USA March No No susoctocog alfa Obizur Baxter Haemophilia A Factor VIII stimulant USA Nov No No suvorexant Belsomra Merck & Co Insomnia Orexin receptor antagonist Japan Nov Yes No tasimelteon Hetlioz Non-24-hour Vanda Pharmaceuti- sleep-wake disorder cals Melatonin 1 receptor agonist / Melatonin 2 receptor agonist USA April No Yes tavaborole Kerydin Anacor PharmaceutiOnychomycosis cals / Novartis Leucyl-tRNA synthetase inhibitor USA Sept Yes No tedizolid Sivextro Dong-A / Cubist Acute bacterial skin and skin structure infections Protein 50S ribosomal subunit inhibitor USA July No No tofogliflozin Apleway Roche / Sanofi Type 2 diabetes Sodium/glucose cotransporter 2 inhibitor Japan May No No turoctocog alfa NovoEight Novo Nordisk Haemophilia A Factor VIII stimulant Germany Jan No Yes umeclidinium bromide + vilanterol**** Anoro Ellipta Theravance / GlaxoSmithKline Chronic obstructive pulmonary disease Long-acting muscarinic antagonist USA April No No vaniprevir Vanihep Merck & Co Hepatitis-C infection HCV nonstructural protein 3 inhibitor / HCV nonstructural 4A inhibitor Japan Nov Yes No vedolizumab Entyvio Takeda Crohn’s disease & ulcerative colitis Alpha4beta7 integrin antagonist / Integrin antagonist USA June No No vorapaxar Zontivity Merck & Co Thrombosis Protease-activated receptor-1 antagonist USA July Yes No Major depressive disorder 5 Hydroxytryptamine 1A/1B/1D receptor agonist / 5 Hydroxytryptamine 3/7 receptor antagonist / 5 Hydroxytryptamine uptake inhibitor USA Jan No No vortioxetine Brintellix Lundbeck / Takeda Source: Pharmaprojects ®, February 2015 *nepupitant is the NAS in this combination **ombitasvir + paritaprevir are both NASs in this combination *** ledipasvir is the NAS in this combination ****umeclidinum bromide is the NAS in this combination 7 Having said that, no one company can truly be said to have had a stellar year. Leading the way in terms of bringing new NASs to market were Novartis, Merck & Co and Eli Lilly, with just four (compared to GlaxoSmithKline’s seven in 2013). These companies head our Table 2, which shows how many NASs each of the Top 10 pharma companies produced, augmented by any other companies which launched two or more. GSK features high up the list again, although 3 NAS launches from a pipeline of >250 drugs is maybe not quite the ROI it would wish to see long-term. One other Top 10 company, Roche, produced three NAS launches, while the two BIs, Boehringer Ingelheim and Biogen-Idec, punched above their weight in also delivering three apiece. Table 2. Top company NAS launch performance 2014 Company Number of NAS launches 2014 Position by pipeline size in Top 25 Novartis 4 2 Merck & Co 4 6 Eli Lilly 4 10 GlaxoSmithKline 3 1 Roche 3 3 Bristol-Myers Squibb 3 11 Boehringer Ingelheim 3 20 Biogen Idec 3 – Sanofi 2 8 Takeda 2 9 AbbVie 2 16 Gilead 2 – Johnson & Johnson 1 5 Pfizer 1 7 AstraZeneca 0 4 Source: Pharmaprojects ®, February 2015, Citeline Languishing at the bottom of this list are three Top 10 companies: Johnson & Johnson, and Pfizer and its intended prey from 2014, AstraZeneca. The latter was the only of the biggest of big pharma companies to launch not a single NAS, although while mounting its defence, it has made much of its late-stage pipeline and the NASs it could deliver in 2015/16. It will certainly need to fire its boosters this year to maintain escape velocity and pull clear of Pfizer if it is going to survive. Meanwhile, relative upstart Gilead is twinkling brightly, expanding its HCV franchise and launching a new leukaemia drug to boot. 8 Talking of cancer, it’s also instructive to segment the year’s new drugs by their primary therapeutic focus, which is what Figure 2 does. This shows that oncology drugs were, as in the previous year, only the third most successful group in terms of numbers of market entrants. This is despite taking by far the biggest chunk of the R&D pipeline and budget. This demonstrates that cancer remains a high-risk area, and it’s interesting to additionally note that seven of the eight new cancer drugs have orphan drug status. High risk then, but high return. Figure 2. 2014 NAS launches by Therapeutic Group 2 2 3 3 2 Alimentary/Metabolic Products 13 Anti-infective Products Anticancer Products Blood and Clotting Products Cardiovascular Products Musculoskeletal Products Neurological Products 7 Respiratory Products Sensory Products 8 21 Source: Pharmaprojects ®, February 2015, Citeline As in 2013, anti-infectives account for by far the largest group of debuting drugs. The difference this year is the contribution to this figure by vaccines. Only six of the 21 anti-infective launches in 2014 were vaccines, compared to ten out of fifteen the year before. So there was a burst of new anti-infective molecules coming to fruition – fuelled in no small part by the explosive revolution in the hepatitis-C market. There were no fewer than seven new drugs for this disease reaching the market; now all the industry has to do is work out how to get them to all of the patients who would benefit from them. Pricing issues are thus far proving restrictive, but as more and better molecules continue to join the first generation of direct anti-HCV antivirals, the situation is in rapid flux. In fact, with most of the drugs approved so far being afforded breakthrough therapy status in some form, the market is evolving so rapidly that the US FDA is now moving to rescind this status from follow-on drugs which were only granted it a little over a year ago. Alimentary/Metabolic was second biggest beneficiary of new drug launches, with thirteen. Type 2 diabetes again led the way here, with its six new molecules coming in the form of four additional SGLT2 inhibitors and two GLP-1 agonists. Neurological, the second biggest therapeutic area in terms of pipeline activity, was once again not very successful in producing new drugs, delivering just three. But there were no new Dermatological or Genitourinary drugs at all. 9 In terms of the countries which hosted market introductions, the USA was as usual far out in front, with 34/61 (56%) of NAS launches occurring there first. This is a slightly smaller proportion than in 2013, but a larger number. The big news this year was Japan’s mighty ten debutants, far exceeding the four seen in the preceding twelve months. This beat the whole of Europe combined. It was interesting to see that, with additional Asian contributions from China, India and Vietnam, the pharma star was certainly rising in the East. Figure 3. 2014 NAS launches by region 3 2 2 3 USA Japan China Other EU Germany 4 India UK 3 34 Other 10 Source: Pharmaprojects ®, February 2015, Citeline The big news this year was Japan’s mighty ten debutants, far exceeding the four seen in the preceding twelve months. This beat the whole of Europe combined. 10 The novel NASs of 2014 – some stellar advances, but some astronomical prices We now move on to highlighting the most exciting new drugs of 2014 – the twelve novel NASs. These are the drugs hitting the market with a mechanism of action which has not been seen in a launched drug before. They break down this year into three anti-infectives, four anticancers, and one apiece for blood/ clotting, cardiovascular, metabolic, musculoskeletal and neurological. Four of these drugs also have the double-whammy of orphan drug status. Let’s start with what seems very much to be the mal du jour in terms of pharma innovation, hepatitis-C. Even in almost thirty years of commenting on pharmaceutical R&D, it is still comparatively rare for me to be able to say in all honesty that the treatment of a disease is undergoing a total revolution. We are not there yet, but replacing an only partially effective and lengthy treatment regimen including an injectable drug with potentially severe side-effects, with an all oral, well tolerated 4-6 week treatment with cure rates approaching 100%, now seems an achievable goal. As previously noted, seven drugs joined the HCV small molecule armoury last year, and of these two were first-in-class. Bristol-Myers Squibb’s Daklinza (daclatasvir) was one of these, becoming the first HCV nonstructural 5A inhibitor on the market when it was first commercialized, in Germany back in August. It is also under Phase II development as part of BMS’s triple fixed-dose combo, whose other constituents are asunaprevir and beclabuvir. It was joined in October by the Japanese launch of (Vanihep) vaniprevir. This molecule has dual activity in inhibiting both the NS3 protease and the nonstructural 4A protein, and it is the latter activity which is the novel mechanism here. This brings the total number of HCV targets hit by direct-acting antiviral small molecules to four. Vanihep is interesting for its preferential activity on genotype 1 only, which is found in two-thirds of Japanese HCV patients; Merck has thus decided to only market the drug in this territory. The third new anti-infective NAS is Kerydin (tavaborole), the first leucyl-tRNA synthetase inhibitor to make it through the development process. It’s an antifungal, indicated in the US for the treatment of the nail infection, onychomycosis. It was developed by Anacor and licensed out to Novartis’ subsidiary, Sandoz. So in infectious disease, viral and fungal diseases benefitted from novelty, but there was no such good news for bacterial infections. The four cancer drugs boasting a novel mechanisms are all also orphan drugs. Ono’s Opdivo (nivolumab) became the first PD-1 antagonist on the market when it was launched in Japan in September. Ono partnered with cancer specialist Bristol-Myers Squibb to bring Opdivo through development, picking up orphan drug status for melanoma in both Japan and the US along the way. Its successful launch meant that it just pipped Merck & Co’s similar molecule Keytruda (pembrolizumab) to the title of first PD-1 antagonist available. The PD-1 (programmed cell death 1) protein is a cell surface protein which is here being targeted by both of these drugs to treat melanoma. As such, these two drugs are among the first market entrants from the super-hot field of anticancer immunotherapeutics. These particular antibodies have broad potential use in other cancers too, and indeed Opdivo has already been filed in the EU and US additionally for non-small cell lung cancer. It was something of the star molecule at November’s AACR-NCI-EORTC Symposium on Molecular Targets and Cancer Therapeutics meeting, held in Barcelona. 11 It is joined in both its novelty and orphan drug status (ODS) by Blincyto (blinatumomab), the first CD19 antagonist to gain approval, having been brought to market by Amgen for the treatment of adults with Philadelphia-negative relapsed/refractory B-precursor acute lymphoblastic leukaemia (ALL). First launch was in the US, where it has ODS and breakthrough therapy status, and it also has ODS in the EU, where it is awaiting approval. CD19 is a cell surface molecule which assembles with the antigen receptor of B lymphocytes and decreases the threshold for antigen receptor-dependent stimulation. The drug is one of three monoclonal antibodies in this set of novel anticancers and seven in the NAS list as a whole in what was a pretty successful year for this class of agents. The third anticancer MAb with a novel mechanism is Lilly and Dyax’s Cyramza (ramucirumab), which acts via the new mechanism of direct antagonism of the VEGFR-2 receptor. The drug has been launched in the US for advanced or metastatic gastric cancer or gastroesophageal junction (GEJ) adenocarcinoma with disease progression on or after prior fluoropyrimidine- or platinum-containing chemotherapy. Zydelig (idelalisib) is the final anticancer, but in this case is not a MAb. It is a small molecule PI3 kinase delta inhibitor, and is in fact the first PI3K (phosphatidylinositol 3-kinase) inhibitor of any type to reach the market. Its US approval for relapsed chronic lymphocytic leukaemia and non-Hodgkin’s lymphoma was swiftly followed by the nod from the EU, and it is another success story for high-flying Gilead. There were two other novel NASs which also had the distinction of being orphan drugs. In the metabolic area, we saw a new molecule for an enzyme efficiency disorder: BioMarin’s Vimizin (eloslfase alfa) for mucopolysaccharidosis IV. This enzyme replacement therapy has distinguished itself by being another new drug to command a very high price – an eye-watering £190,000 per year. But with there only being around 3,000 MPS IV patients in total in the developed world, this is the very definition of a rare disease, and with Vimizin being the only therapeutic drug option, the cost is at least understandable. The final orphan is Translarna (ataluren) from PTC Therapeutics, which was launched first in Germany for the treatment of patients with nonsense mutation Duchenne muscular dystrophy. Mechanism-wise, this is an interesting one. The drug was presumed to act by promoting read-through nonsense mutations in mRNA; however, it has been suggested that the ascription of this mechanism was due a misinterpretation of a preclinical result. Nevertheless, it appears to increase dystrophin levels, and received conditional approval in the EU. It remains in Phase III for another genetic disorder involving nonsense mutations, cystic fibrosis. For now at least, it is recorded as the first ribosomal read-through stimulant to reach the market. CNS is an area seemingly hitting something of an impasse at the moment. Despite being the second biggest therapy area in terms of pipeline, it is showing the lowest growth rate, and has struggled in recent years to deliver many new compounds. However, one of its three NASs this year did at least exhibit some novelty. Merck & Co had success in Japan again, here with Belsomra (surovexant), a new insomnia drug and the first orexin receptor antagonist. It has since also been launched in the US. 12 Finally, there were two first-in-class NASs in the clotting/cardiovascular area. Zontivity (vorapaxar) is another new drug from Merck & Co. It is the first protease-activated receptor-1 antagonist; other drugs with this activity, such as Eisai’s atopaxar, fell along the wayside to leave Merck as the sole inhabitant of this space. It received the green light in the US for use as an antithrombotic in patients with a history of heart attack or with peripheral arterial disease and no history of transient ischaemic attack or stroke. Lastly, we have Reasanz (serelaxin) from Novartis for heart failure; the first relaxin. It has been launched in Russia, but regulatory authorities rejected its first applications in both the EU and the US. Resubmissions are planned following receipt of results from trials which are currently running, but it would be fair to say that the jury is still out on this one. This constellation of first-in-class drugs illuminates Merck & Co as the most successful at delivering novelty through 2014, with three drugs. This just eclipses Bristol-Myers Squibb, which had two. This constellation of first-in-class drugs illuminates Merck & Co as the most successful at delivering novelty through 2014, with three drugs. This just eclipses Bristol-Myers Squibb, which had two. 13 Orphan drugs put on spectacular display As well as novel drugs and breakthrough therapies, there was more evidence in 2014 of the industry’s current fascination with orphan drugs and the high prices which such niche products can command. Leaving aside the aforementioned pricing issues with the new generation of hepatitis-C drugs (which, let’s not forget, is nothing like an orphan disease in terms of prevalence), the year saw a historically high and potentially budget-busting total of 22 NASs launched which have orphan drug status granted in at least one market. The previous year, there were only twelve, and thus the proportion of NASs which are orphan drugs has risen from a quarter to over a third. But for the singular feature which really sets 2014 up as historic, we have to go back to that very first graph in this report. The pharma R&D universe has been steadily and continuously expanding for many years, but until now, growth in the number of drugs reaching the market has eluded us. Last year though, this finally seems to have arrived. The supernova-like explosion of 61 new active substances onto the market in a single year is completely without precedent, and both exceedingly welcome, and probably a little surprising too. The birth of a new epoch, or a flash in the pan? After all, supernovae are notorious for burning brightly and briefly before collapsing, often into black holes. I don’t think that will happen, but the danger is always that if such a spectacular result is not repeated in 2015, the industry’s mood might at least darken. For now, let’s just bask in the glow of record-breaking results. Pharmaprojects will be continually observing the pharma universe as always, watching for more signs and portents. The supernova-like explosion of 61 new active substances onto the market in a single year is completely without precedent, and both exceedingly welcome, and probably a little surprising too. The birth of a new epoch, or a flash in the pan? 14 15 www.citeline.com [email protected] Citeline provides the world’s most comprehensive and United States 52 Vanderbilt Avenue 11th Floor New York NY 10017 USA +1 646 957 8919 +1 888 436 3012 reliable real-time R&D intelligence to the pharmaceutical United Kingdom Christchurch Court 10-15 Newgate Street London EC1A 7AZ United Kingdom +44 20 7017 5000 Japan Kotakudo Ginza Building, 7th Floor 5-14-5 Ginza Chuo-ku Tokyo 104-0061 +81 351 487 670 China 16F Nexxus Building 41 Connaught Road Hong Kong +852 3757 9007 Australia Level 7 120 Sussex Street Sydney NSW 2000 +61 2 8705 6900 Citeline © 2015. All rights reserved. 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