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.
UnaCom Pharma News
Monday 15th April
Roche receives EMA approval for Hemlibra label
The EMA (European Medical Agency) has approved Hemlibra for the use in the majority of patients with haemophilia A, extending its use beyond the minority who has developed resistance to factor Vlll drugs. Roche says that the now ‘extended’ label will be suitable for all age groups and can be used at multiple dosing options eg once weekly, every two weeks etc. This patient friendly dosing schedule is proving popular with patients, who are used to having to inject factor Vlll replacement drugs multiple times per week.
Hemlibra is used to reduce the frequency of bleeding episodes in children and adults with haemophilia A. Hemlibra is a bispecific antibody that mimics the role factor Vlll plays in the blood.
Tuesday 16th April
Sanofi replaced by Regeneron as R&D partner for Alnylam
Regeneron has agreed to pay $800m to gene silencing specialist Alnylam, to buy into its pipeline of drugs for CNS, eye diseases and liver diseases. This deal was only recently announced after Sanofi and Alnylam dissolved a rare disease R&D partnership set up in 2014.
The $800m, will be split into a $400m, upfront payment as well as a $400m on a 5% equity stake in the biotech. There is also the potential added payment of $200m in milestones if the drugs hit certain early clinical development targets.
The specific of the deals does not cover any specific programmes for CNS or eye diseases, as the intention of this collaboration is to combine their respective strengths. Regeneron will take the lead in the eye disease category, as it already has a strong blockbuster drug, with Alnylam already signed up for royalties and milestone payments.
The liver disease alliance will combine both companies existing pipelines to a multi-drug regimen for diseases including non-alcoholic steatohepatitis (NASH), a promising target for the biopharma industry.
This deal is interesting because it is a deal between two biotech companies rather than the common biotech/big pharma model. This further establishes the emergence of RNAI as a new drug class after decades of development.
Wednesday 17th April,
Novartis releases data to further support case for gene therapy Zolgensma in SMA
Novartis released data from its phase 3 trial of the newly acquired gene therapy Zolgensma, with the data reinforcing the drugs already impressive efficacy profile. This data comes ahead of a decision to made by the FDA to evaluate the drug for the treatment of the most severe form of spinal muscular atrophy (SMA).
Zolgensma has been under review since December, after it was given a priority status, and fast tracked by the FDA.
The most promising results from the phase showed that the drug helped those with SMA type 1 sit unassisted for at least 30 seconds. This milestone was achieved for three patients back in phase 1 in September but since an extra five patients have displayed similar results.
With a high chance of approval by the FDA, there has been a focus on the pricing of the treatment. Novartis has already suggested that the one-time treatment be priced at $4m per patient but US medicine pricing watchdog ICER, has urged Novartis for a fair pricing agreement. This comes after current SMA treatment by Biogen, being classed as overpriced, with Spinraza being on the market since 2016 coming in at a price of $750,000 for the first initial year and $375,000 per year thereafter. ICER stated that the price should be slashed by at least 82.76% for the first year and then should cost between $36,000- $65,000 per year thereafter.
ICER has called for Zolgensma to be priced somewhere between $900,000 and $1.5m. Novartis will not release any pricing agreements until the product has been approved.
Thursday 18th April,
Roche announces ‘discovery proteomics’ could be heading for clinical trials
Proteomics is a technology that can take a snapshot of protein expression in tissue samples, this technology is already used in drug discovery and could soon be applied to clinical trials states Roche.
The quantification of proteins in biological samples could be used to identify novel biomarkers and to generate additional information to help explain why clinical intervention is or is not working in a particular patient.
Roche has been working with proteomics specialist Biognosys on a mock trial to explore how it could be used clinical trials. A study has been performed by the two companies looking at cancer samples taken from clinical trials. The method proved to be quick as it uses data analysis to compare the signatures against a database of references. Almost 9,000 proteins were identified in the study with researchers saying that the results had significant statistical power to ‘detect subtle changes in protein expression that may occur in tumor tissue’
Friday 19th April
Novo Nordisk and Gilead Sciences to collaborate to target NASH.
They are too collaborate on new combinations targeting NASH ( Non-alcoholic Steatohepatitis) using Novo’s diabetes drug semaglutide. The drug will be combined with Gilead’s cilofexor and firsocosat in a proof of study concept, as they are both investigational molecules with no approvals of yet.
This is a smart move by both companies in a crowded field of late stage pipeline contenders. Gilead announced its clinical trial results investigating cilofexor with firsocosat in those with NASH, with promising results of out of the 20 patients treated , 74% of them experienced a significant decline of at least 30% in hepatic fat, a biomarker of the disease.
NASH is a chronic liver disease characterized by fat accumulation and inflammation in the liver , which can lead to scarring and fibrosis that impairs liver function.
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