The FDA approved 22 novel drugs in 2016 – a notable decline versus recent years. FirstWord takes a closer look at the 13 new product approvals which generated the most headlines last year…
Merck & Co.'s return to the hepatitis C market occurred too late to allow significant inroads to be made into Gilead Sciences' dominance of this segment. Nevertheless, Zepatier – approved by the FDA in January – forms the basis of a multi-product franchise that is expected to deliver Merck blockbuster revenues over the next few years, despite the need to compete aggressively on price. Speaking on the company's most recent quarterly earnings call, management suggested that it has won a number of contracts in the US market, which should bolster performance of the brand in 2017. More recently, Merck was awarded considerable royalties in an ongoing patent lawsuit with Gilead.
In March, Eli Lilly's Taltz became the second IL-17a inhibitor to secure US approval for moderate-to-severe plaque psoriasis (following approval of Novartis' Cosentyx in 2015). Cosentyx has enjoyed a stellar launch, which means that while Taltz may struggle to compete directly with Novartis' brand, there is considerable slipstream for Eli Lilly's product to ride in.
Analysis: Physician Views Poll Results: Cosentyx growth unlikely to be significantly interrupted by launch of Eli Lilly's Taltz and Cosentyx and Taltz offer 'quantum leap' in psoriasis but payers will embrace biosimilars
Teva's Cinqair became the second anti-IL-5 monoclonal antibody to be approved by the FDA for the treatment of severe asthma, when it was greenlighted by the agency in March. GlaxoSmithKline had previously secured first-to-market status for its competing drug Nucala, which was approved in late 2015. According to experts, however, Teva's biggest disadvantage stems from Cinqair's intravenous formulation. Nucala – and a number of other biologics currently in late-stage development for severe asthma – are administered via more convenient subcutaneous injections.
Analysis: ViewPoints: Teva secures FDA approval for Cinqair, but faces tough competitor in GlaxoSmithKline's Nucala, say experts and Physician Views Poll Results: Feedback from 84 pulmonologists suggests AstraZeneca's benralizumab is well positioned, but questions remain over size of market
One of the highpoints in innovation regarding 2016's list of novel drug approvals, AbbVie and Roche's Venclexta has been lauded as a potential game-changer for the treatment of chronic lymphocytic leukaemia (CLL) by key opinion leaders. Initial FDA approval in April occurred in a subset of CLL patients with a specific chromosomal abnormality. Experts are confident, however, that Venclexta will deliver positive data in subsequent studies and become an important future therapy for CLL; in the process building out AbbVie's position in the haematological cancer market.
Analysis: Physician Views Poll Results: US oncologists impressed with AbbVie, Roche's Venclexta in CLL and Physician Views Poll Results: Oncologist feedback indicates AbbVie and Roche's Venclyxto is well placed for EU launch
A notable success story in the CNS field, Acadia Pharmaceuticals' Nuplazid was approved for the treatment of Parkinson's disease psychosis in April, prompting speculation the company could be acquired by one of the sector's large-cap players. Hopes that Nuplazid can also effectively treat psychosis in Alzheimer's disease patients remain in place despite mixed Phase II top-line data released by Acadia last month. Additional data for Alzheimer's disease agitation is expected to read out this year.
Analysis: ViewPoints: Pimavanserin’s success in Alzheimer’s psychosis could be unexpected upside – but extenuating circumstances abound, ViewPoints: Acadia’s Nuplazid launch will require an outside-the-box approach and KOL Views: Expert disagrees with black box for Acadia’s Nuplazid – but agrees the risk is real
Roche became the third company to launch a PD-(L)1 inhibitor when it secured FDA approval for Tecentriq in second-line bladder cancer in May, with positive Phase II data in first-line patients also presented this year. Tecentriq has subsequently been approved by the agency as a therapy for second-line non-small-cell lung cancer (NSCLC) patients irrespective of PD-L1 biomarker status. Roche is due to release much anticipated clinical data in the second half of 2017 from studies assessing the combination of Tecentriq plus chemotherapy in first-line NSCLC.
Intercept Pharmaceuticals' Ocaliva was approved by the FDA in May for the treatment of primary biliary cholangitis (PBC); providing a commercial debut for a product the company hopes to subsequently bring to market for the much more lucrative indication of nonalcoholic steatoheptitis. Sales in PBC to date have been muted.
Gilead secured FDA approval for the first pan-genotypic hepatitis C therapy in June when Epclusa was green-lighted by the agency. The company surprised analysts by pricing the drug at a discount to both Harvoni and Sovaldi in the US market. However, although stronger uptake was seen in the third quarter, Gilead's overall hepatitis C sales continue to decline at a faster rate versus sell-side consensus, which is exerting pressure on management to deliver transformational M&A.
Analysis: Physician Views Poll Results: 177 infectious disease doctors assess newly approved Epclusa and clinical hold of Regulus’ RG-101 and ViewPoints: Gilead’s pricing of new HCV drug again draws gasps, albeit of a different nature – lesson learned?
Approved by the FDA in July for the treatment of dry eye disease (DED), Shire's Xiidra could become the company's best-selling product of all time, argue some analysts, who have been quick to ascribe blockbuster revenue projections. Early signs are positive, based on initial prescription trends and feedback from ophthalmologists, with considerable growth likely to come from Shire's efforts to expand the overall DED market. While Allergan's established therapy Restasis is therefore likely to lose some market share, it too will remain a sizable product in the US.
The most controversial FDA new drug approval of all time? Certainly among the class of 2016. Sarepta Therapeutics' Duchenne muscular dystrophy (DMD) treatment Exondys 51 was approved by the agency in September after a protracted regulatory process notable for the prominent role of patient advocacy. With clinical evidence to support approval extremely limited, some US insurers have been hesitant to cover the drug, which is expected to slow the launch of Exondys 51. This could prove to be an incentive in discouraging other companies from pursuing approval strategies that utilise patient advocacy as a means to compensate for very small clinical data sets; a concern that has arisen as a result of the FDA approving Sarepta's drug.
FDA approval of Eucrisa (a month earlier than expected in December) may be viewed as validation of Pfizer's post-Allergan acquisition strategy. The US giant inherited the mild-to-moderate atopic dermatitis treatment when it acquired Anacor Pharmaceuticals for $5.2 billion in June. Pfizer management has pinned its hopes on peak Eucrisa sales reaching $2 billion to more than justify the deal. Whether the topical therapy can meet these expectations may be largely dependent on how payers manage access to Eucrisa.
Having failed spectacularly to secure approval for rociletinib (in certain types of NSCLC) Clovis Oncology regained some much needed credibility when the FDA approved Rubraca as a third-line treatment for BRCA mutation ovarian cancer last month. Commercially speaking, however, adoption of Rubraca may be limited by AstraZeneca's already marketed Lynparza and pending approval of Tesaro's rival PARP inhibitor in second-line patients.
Analysis: ViewPoints: New rucaparib data prick hole in Clovis’ M&A-fuelled valuation – bargain buy or caveat emptor?, ViewPoints: AstraZeneca hints at impressive data for its ovarian cancer drug Lynparza and KOL Views: PARP inhibitors here to stay, says leading oncologist
The FDA's last approval of 2016, which offers further evidence of the agency's willingness to grant rapid access to breakthrough therapies. Biogen and Ionis Pharmaceuticals' Spinraza – for the treatment of spinal muscular atrophy (SMA) – has emerged rapidly over the past six months with earlier readout of positive pivotal-stage clinical data pre-empting US approval. FDA clearance has not only come early, but with a broad label facilitating use in all subtypes of SMA. Biogen and Ionis have sparked some controversy, however, by announcing a much higher-than-expected price.
Analysis: KOL Views: Nusinersen’s success is watershed moment for SMA research though diagnostic innovation will be key to commercial success, according to leading expert and KOL Views: Despite recent bobbles, RNA therapeutics space poised for a big couple of years, says leading researcher
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