At the time of writing the FDA has approved 44 novel drugs in 2017 (the agency’s website has a list here); throw in a handful of biologic approvals (here) and the 46 approvals cleared in 2015 (a 66-year high) has already been beaten. With the FDA on a roll, there is every chance the total could increase further before year-end. In the meantime, we took a closer look at twelve of the most important new approvals in 2017…
Dupixent (atopic dermatitis) – Regeneron Pharmaceuticals/Sanofi
Key opinion leaders are confident this biologic therapy will shift the treatment paradigm for moderate-to-severe atopic dermatitis; in the process setting a notable benchmark for subsequent products to beat (the competition is emerging). Analysts have spent much of 2017 tracking speed of adoption and evaluating future opportunity in other indications, following US approval in AD in March. Earlier this year, Regeneron Pharmaceuticals and Sanofi released positive Phase III data in severe asthma, where Dupixent has been studied in a broader population versus competitor biologics. With more data to come, both companies maintain that Dupixent is a 'pipeline within a product,' prompting the question as to whether it can scale similar commercial heights to AbbVie's Humira.
Hemlibra (haemophilia A) - Roche
Recently approved in the US for the treatment of haemophilia A patients who have developed inhibitors to Factor VIII therapy, the once-weekly bispecific antibody Hemlibra has also demonstrated a "significant and clinically meaningful," reduction in treated bleeds in non-inhibitor patients. Roche looks poised to be a disruptive force in both these markets, particularly the former (where it has been shown to reduce bleeding rates by 87 percent versus patients who didn't receive prophylactic therapy), and will provide a new competitive threat to the likes of Shire and Novo Nordisk thanks to a potent combination of superior efficacy and greater convenience. Consensus forecasts suggest sales will edge towards $2 billion by 2022, despite some safety concerns. Shire is not taking the threat lightly.
Further analysis - KOL Views Results: Leading haematologist outlines expectations for Hemlibra's launch trajectory and Physician Views Poll Results: On balance, Roche's Hemlibra poised to transform haemophilia A market.
Kymriah (acute lymphoblastic leukaemia) – Novartis | Yescarta (diffuse large B-cell lymphoma) - Gilead Sciences
Novartis' Kymriah became the world’s first marketed CAR-T product when it was approved by the FDA in August, for previously treated paediatric and adolescent patients with acute lymphoblastic leukaemia. Novartis launched Kymriah with a pledge; if a patient doesn't respond to therapy after a month they don't pay (it has a list price of $475,000). Pivotal-stage data shows that among 63 evaluable patients, 83 percent had achieved complete remission at three months. Novartis is now seeking approval in diffuse large B-cell lymphoma (DLBCL).
Assuming Kymriah is approved in DLBCL, it will compete with Yescarta, which was approved by the FDA in October. Developed by Kite Pharma, the biotech was acquired by Gilead Sciences for $11.9 billion shortly before Yescarta was approved. Clinical data for both products look remarkably similar and increasingly durable; suggesting that other factors – such as toxicity and logistical support – could prove critical in determining commercial success and competitive dynamics. Gilead's outlay raises the stakes; recent reports suggest that CAR-T will be a long-term play.
Further analysis: KOL Views Results: Leading oncologist says CAR-T therapies look more similar than different in DLBCL and Physician Views Poll Results: Kymriah’s toxicity and outpatient use could prove critical in showdown with Yescarta.
Luxturna (retinal dystrophy) - Spark Therapeutics
FDA approval of Spark Therapeutics’ Luxturna in mid-December came as no surprise (it was backed unanimously by an advisory committee in October) but is a historical event; cleared as a treatment for an inherited form of retinal dystrophy, Luxturna becomes the first bona fide gene therapy to be approved in the US. Specifically it is the first directly administered therapy authorised in the US that targets a disease caused by mutations in a specific gene. As a one-time treatment, what will Luxturna cost? – Spark plans to disclose its pricing strategy in early 2018. Analysts predict it could cost $1 million but Luxturna is not a cure; in some patients it has been shown to halt disease progression and restore some functional vision. Those patients who respond to therapy typically show signs of this within a month of treatment.
Ocrevus (multiple sclerosis) - Roche
Approved by the FDA in March, consensus forecasts model 2022 sales of more than $4 billion for Ocrevus. Roche's drug has received glowing praise from key opinion leaders and dominated a recent snap-poll of neurologists we ran to benchmark current and future use of multiple sclerosis therapies. Early sales growth has been strong and first year revenues will come close to achieving blockbuster status. Marketed for relapsing remitting and primary progressive MS (as the first approved therapy), experts suggest that Ocrevus is also being used off-label in secondary progressive patients. Roche also deserves kudos for its pricing strategy, with Ocrevus launched at a notable list price discount versus major competitors in the US market.
Further analysis - KOL Views Results: Leading neurologist says fears about Ocrevus’ PML risk are overblown and Physician Views Poll Results: Prescriber feedback on initial Ocrevus use supports key KOL assertions; Tysabri baring initial brunt of new competition.
Ozempic (diabetes) - Novo Nordisk
Novo Nordisk has been clear; Ozempic - until recently known only as semaglutide - will be its most important product over the next five years and possibly beyond. With recent FDA approval, the Danish diabetes specialist makes its debut in the once-weekly GLP-1 agonist market and will look to build on the overall leadership of this drug class it holds through the once-daily therapy Victoza; in the process heading off the competitive threat posed by Eli Lilly's Trulicity. Approval in diabetes could be just the start for Ozempic; Phase III data for Novo Nordisk's oral version of semaglutide (key opinion leaders argue this will be a true game changer) are due next year and will coincide with the start of a large pivotal-stage study programme for Ozempic in obesity.
Rydapt (acute myeloid leukaemia) – Novartis | Idhifa (AML) – Celgene/Agios
2017 has been a breakthrough year for acute myeloid leukaemia (AML) therapies. For decades, the mainstay of treatment has been cytarabine-based chemotherapy regimens and a number of other cancer therapies used on an off-label basis. In April, Novartis’ Rydapt was approved for newly diagnosed AML patients who carry the FLT3 genetic mutation.
In August, Celgene and Agios’ Idhifa was approved for relapsed AML in patients with a mutation of the IDH2 gene
Shingrix (shingles vaccine) - GlaxoSmithKline
GlaxoSmithKline has secured approval of two new products in recent months, both of which will shape near-term assessment of the company under new stewardship of CEO Emma Walmsley (the other being its 'closed triple' combination therapy Trelegy Elipta for COPD). Shareholders would like to see GlaxoSmithKline revive its pharma division, but it is a vaccine that could give the company a near-term boost. Commercial positioning of Shingrix has been "greatly strengthened" by the US Advisory Committee on Immunization Practices' (ACIP) decision to recommend it as a preferred vaccine over Merck & Co.'s Zostavax, argue analysts at Bernstein. They expect global sales to reach around $1.7 billion by 2022, although more will expected of the company, particularly given recent management hires.
Tremfya (psoriasis) - Johnson & Johnson
Concerned, perhaps, by the competitive threat posed by Novartis' Cosentyx and Eli Lilly's Taltz (both IL-17 inhibitors), Johnson & Johnson successfully accelerated US approval of Tremfya with a priority review voucher. Johnson & Johnson's Stelara is already a firm favourite among dermatologists and Tremfya offers better selectivity, targeting only the IL-23 receptor rather than both IL-12 and IL-23. This means lower toxicity, while pivotal data have shown superior efficacy versus both Stelara and AbbVie's Humira. Less frequent dosing is another benefit, but the challenge may be overcoming physicians' real-world experience with Stelara, alongside the mechanistic optionality provided by Cosentyx and Taltz. Nevertheless, blockbuster status beckons for Tremfya in a crowded market.
Further analysis: ViewPoints: Bar set high for AbbVie, Boehringer Ingelheim's push into psoriasis, Physician Views Poll Results – Tremfya may have raised the bar again, but competition is more intense and Physician Views Poll Results: Dermatologist feedback showcases Johnson & Johnson's balancing act with Tremfya.
Zejula (ovarian cancer) - Tesaro
PARP inhibitors have remained a topic of hot debate in 2017 (among oncologists and investors alike) and Tesaro's Zejula set two new benchmarks when it was approved by the FDA in March; as the first drug in class labelled with no requirement for biomarker testing and the first PARP to be approved for maintenance use in ovarian cancer patients who have previously responded to treatment with chemotherapy. Tesaro has been widely speculated on as a potential acquisition target, but such a deal is yet to materialise, while competition within this drug class remains intense.
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