Orphan Drugs Market to soar to $224bn by 2024
Orphan drugs are drugs that are intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions. They are drugs that are not developed by the pharmaceutical industry for economic reasons, but which respond to public health needs.
The pharma analysis group, Evaluate Pharma, say breakthrough treatments such as Novartis’ Zolgensma and bluebird bio’s Lentiglobin will contribute to a 12% compound annual growth rate for orphan drugs through to 2024. Non-orphan drugs are also expected to grow by 2024, but only by 6% each year. Cell/gene therapy treatments are leading the way for orphan drugs, with seven of the top 20 R&D orphan products by net present value are either a cell or gene therapy.
The report also revealed several interesting developments, with Celgene set to topple Novartis as the world’s biggest orphan drug developer as its Revlimid, multiple myeloma therapy, is predicted to be the leading orphan drug product in 2024. It also predicted that the current trend of Big Pharma acquiring smaller companies for its orphan drugs will continue M&A deals, which has been supported by recent deals such as Roche’s $4.8bn acquisition of Spark and Novartis $8.7bn buy-out of AveXis.
Tuesday 23rd April
Biotech industry now a core component of the pharma industry, but its success is wavering
The pharma industry can now be called the biopharma industry, with new data showing that biotech/emerging companies developing the majority of the drugs approved in 2018. This was revealed by a new study of industry trends from IQVIA, The changing landscape of Research and Development. It found that emerging Biopharma companies approved and registered 47% of new drugs, while Big pharma only had 25% patented. This shows a trend in the medicine development that Big pharma are stuck in a position where they have to depend on external innovation.
The report reveals that 2018 was a record-breaking year in terms of new medicines reaching the market with 59 novel treatments reaching the US alone. With almost half of the drugs having orphan drug status and over a third were identified as first in class therapies.
However overall sector productivity, including return on investment from its R&D spending, does not paint an optimistic picture. The report found a drop in the success rate of clinical development from phase 1 trials to regulatory submission, with the percentage of drugs successfully passing to the next stage of development falling to 11.4% in 2018 down from 14.4% in 2017 and below the average 14% over the last decade. IQVIA found rising failure rates in phase 1 as the biggest problem. Decline in phase 1 activity could be linked to an increase in trial complexity which includes number of trial participants, eligibity criteria, research sites countries and endpoints)
Over the next five years, IQVIA predicts that improvements in trial productivity will be driven by a handful of key trends such as biomarkers, pre-screened patient pools and predictive analytics.
Wednesday 24th April
Roche’s, Hemlibra off to a fantastic start as Roche raises 2019 forecast
The expected decline of Roche’s ‘ big three’ antibody drugs has yet to arrive, amid new product sales offsetting the declines in the first quarter.
There was some decline in their portfolio, with immunology drug MabThera and Herceptin for breast cancer seeing declines in Europe where they fell 38% and 44% respectively. But this was offset by their new products such as Ocrevus for M.S, cancer immunotherapy Tecentriq and new haemophilia drug Hemlibra.
Roche’s third megabrand, cancer antibody Avastin grew 9% ahead of the start of biosimilar competition due this year in the US.
Overall group sales rose 8% to $14.71bn with pharma revenues up 10% on strong demand for the new crop of medicines, as such Roche has raised its 2019 outlook. Ocrevus sales came in at 836m CHF in the quarter, up 2/3 on the same period last year, continuing Roche’s most successful launch ever. New launch, Hemlibra is building momentum with sales at 219m CHF, making almost as much in this quarter as it did in 2018 after approval in a much larger patient population.
Roche has some exciting prospects in the future with its pipeline set to deliver potential blockbusters this year, with CEO Schwan confident that the company will be able to grow despite the impact of biosimilars on its three leading products. There are also exciting prospects in Roche hoping to complete its acquisition of gene therapy specialist Spark Therapeutics and its new blindness treatment Luxturna.
Thursday 25th April
Approval by FDA for GSK Dovato in HIV treatment
The FDA has approved the two-drug single tablet regimen (STR) HIV treatment Dovato. It is the first ever once a day two drug STR for treating patients. The thought behind this treatment is that by giving patients a simpler regimen containing fewer drugs, the expected long-term management of the condition will be easier, with patients developing fewer side effects.
This opens a new front in the battle in supremacy in this field, with predictions that this will become a blockbuster drug and it will be able to challenge GSK’s top rival in this field of Gilead Sciences. Recently GSK also presented exciting data from its phase three trial for another potentially practice changing treatment. A once a month HIV treatment that could free people with the virus from having to take daily treatment
Friday 26th April
Gileads NASH drug fails phase three test
Gilead has fallen at a hurdle to become the first to market non-alcoholic steatohepatitis (NASH) after one of its key candidates failed a key phase three test.
During the STELLAR-3 trial the key inhibitor failed to meet its primary endpoint, which was to improve fibrosis. Gilead also experienced very similar news new months ago, where another indicator selonsertib failed another phase three test.
John McHutchison, chief scientific officer and head of research at Gilead said Gilead will remain focused and committed to developing highly effective treatments for patients living with advanced fibrosis due to NASH.
Although this is a disappointing setback for Gilead, they have not given up on NASH and have other assets in the pipeline due to various acquisitions including Arresto, Pfenex and Nimbus Apollo.
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