Latest news with #Koselugo
Euractiv
6 days ago
- Health
- Euractiv
Bulgarian court ruling widens access to lifesaving drugs
In a significant legal development, Bulgaria's Supreme Administrative Court has overturned a decision by the National Health Insurance Fund (NHIF) regarding a patient with a rare disease's access to medication, opening the door for patients to receive life-saving medications not included in the country's Positive Drug List (PDL). The ruling could have far-reaching implications for people suffering from rare diseases or cancer who are currently excluded from reimbursement due to regulatory loopholes. Dr Antoaneta Toncheva, geneticist and member of the Bridges Community Association, welcomed the news. 'This is the best news I've had in weeks! A small-big step that I hope triggers a landslide of change,' she remarked. Reimbursement obstacles For medicines to be reimbursed, they must be on the so-called 'positive list'. The list determines which medications are fully or partially covered by the state, outlining conditions for prescription, including eligible diagnoses, prescribing specialists, dosages, pricing, and reimbursement levels. Medications not on this list are generally excluded from public funding. However, it is not the only barrier to access. A 2019 ministerial regulation stipulated that only patients under 18 or those who began treatment before reaching adulthood were eligible for reimbursement. Landmark case The court ruling concerns a 21-year-old man diagnosed with a rare genetic disease, Neurofibromatosis Type 1 (NF1). This is a genetically rare condition characterised by the development of multiple tumours. They are not malignant, but they have the potential to become so eventually. The state Health fund had denied funding for his treatment with Koselugo (INN Selumetinib), arguing that under a 2019 ministerial regulation, only patients under 18 or those who began treatment before reaching adulthood were eligible for reimbursement. However, the court found this regulation to be incompatible with Bulgaria's Health Act, which stipulates that treatment outside the scope of mandatory health insurance must be provided under conditions set by the Minister of Health. The judges ruled that the law takes precedence over the ministerial ordinance. Important precedent Expert testimony in the case highlighted that without treatment, the patient risks severe disability. Koselugo blocks specific proteins involved in tumour growth and is expected to reduce the size of plexiform neurofibromas. The court concluded that denying access to the drug based solely on age violated constitutional principles of equality, proportionality, legal certainty, and the protection of legitimate expectations. 'The Constitution obliges the state to protect the health of all citizens without age discrimination,' the ruling states. The judges emphasised that the goal of public health regulations is to ensure quality and accessible healthcare for all. All insured individuals are entitled to free medical assistance, including prescription and access to approved medications, medical devices, and specialised dietary products for home use. Therefore, the NHIF director should have applied the law directly rather than relying on the ministerial ordinance, the court argued. 'The fact that the patient is over 18 does not make him any less in need of state-funded medical care,' the ruling reads. Lawyer Maria Sharkova, who represented the patient, told Euractiv that the ruling sets a precedent for others in similar situations. 'Any patient in need of treatment with a drug outside the list, if it is proven safe and effective, can now rely on this court decision,' she explained. Access improved via courts At the end of 2024, Bulgaria's Commission for Protection against Discrimination concluded that the state systematically discriminates against patients with rare diseases and cancer. The decision followed a year-long campaign by the Bridges Community Association. Although the first court ruling upheld the Commission's findings, the Parliament and the Ministry of Health appealed rather than address the legal shortcomings. The case is now awaiting a final decision from the Supreme Administrative Court. This is not the first time the judiciary has expanded access to healthcare in Bulgaria. In 2024, the Constitutional Court struck down the core financial limits on hospital treatment imposed by the National Health Insurance Fund. As a result, the national healthcare budget increased by 15% within a year, reaching €4.5 billion. Despite this increase, experts warn that the current budget may still fall short of covering the growing healthcare demands triggered by recent legal rulings. [Edited by Vasiliki Angouridi, Brian Maguire]
Yahoo
24-04-2025
- Business
- Yahoo
Merck Q1 Earnings Coming Up: Should You Buy the Stock Now?
Merck MRK will report its first-quarter 2024 earnings on April 24, before market open. The Zacks Consensus Estimate for first-quarter sales and earnings is pegged at $15.48 billion and $2.16 per share, respectively. Earnings estimates for Merck for 2025 have declined from $9.01 to $8.96 per share over the past 30 days. (Find the latest earnings estimates and surprises on Zacks Earnings Calendar.) Image Source: Zacks Investment Research The healthcare bellwether's performance has been solid, with the company exceeding earnings expectations in each of the trailing four quarters. It delivered a four-quarter earnings surprise of 4.68%, on average. In the last reported quarter, the company delivered an earnings surprise of 1.78%, as seen in the chart below. Image Source: Zacks Investment Research Merck has an Earnings ESP of -2.04% and a Zacks Rank #3 (Hold). You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. Per our proven model, companies with the combination of a positive Earnings ESP and a Zacks Rank #1, #2 (Buy) or #3 have a good chance of delivering an earnings beat. You can uncover the best stocks to buy or sell before they're reported with our Earnings ESP Filter. Merck's top-line growth in the first quarter is likely to have been driven by cancer drug Keytruda, as in several previous quarters, aided by additional indications and patient demand. In oncology drugs, Keytruda sales are likely to have been driven by rapid uptake across earlier-stage indications globally, particularly early-stage non-small cell lung cancer. Continued strong momentum in metastatic indications is also likely to have boosted sales growth. The Zacks Consensus Estimate for Keytruda's sales is $7.55 billion, while our estimate is $7.82 billion. In the fourth quarter, U.S. sales of Keytruda benefited from approximately $200 million of wholesale inventory buy-in, which is expected to have reversed in the first quarter of 2025. Higher alliance revenues from Lynparza, driven by increased demand, may have boosted oncology sales. Please note that Merck markets Lynparza in partnership with AstraZeneca AZN. Merck has a profit-sharing deal with AstraZeneca to co-market Lynparza and Koselugo. AstraZeneca and Merck's Lynparza is approved for four cancer types, ovarian, breast, prostate and pancreatic. Lynparza is also being evaluated in combination with Keytruda in late-stage studies for lung cancer indications. Alliance revenues from Lenvima may have also boosted oncology sales. Sales of new drug Welireg are likely to have benefited from the increased uptake for the additional indication of previously treated advanced renal cell carcinoma in the United States. With regard to the HPV vaccine, Gardasil, ex-U.S. sales are expected to have been hurt by lower demand in China due to unfavorable economic conditions. Sales are likely to have increased in the United States due to higher pricing and demand. The Zacks Consensus Estimate for Gardasil is $1.33 billion, while our estimate is $1.28 billion. In the hospital specialty portfolio, generic competition in certain ex-U.S. markets, mainly Europe and the Asia Pacific region, is likely to have hurt sales of neuromuscular blockade medicine — Bridion injection. However, higher demand and pricing are expected to have benefited U.S. sales. The Zacks Consensus Estimate for Bridion is $422.7 million, while our estimate is $410.0 million. Lower demand and pricing in the United States and generic competition in certain international markets, mainly Europe and Asia Pacific, are likely to have hurt sales of the diabetes franchise (Januvia/Janumet). New pulmonary arterial hypertension (PAH) drug Winrevair is likely to have contributed to sales growth with most sales coming from the U.S. market as the company is steadily adding new patients. Another new vaccine, Capvaxive, is also off to an encouraging start in the United States. Investors will be keen to know the sales numbers of Capvaxive, which was approved in the European Union toward the end of March. The Zacks Consensus Estimate for Merck's Pharmaceutical unit is $13.64 billion, while our estimate is $13.82 billion. On the fourth-quarter conference call, the company had guided that Medicare Part D redesign is expected to hurt sales of Winrevair and oncology products, including Welireg, Lynparza and Lenvima in 2025. An update is expected on the first-quarter conference call. In the Animal Health franchise, growth in both companion animal and livestock products driven by higher demand and pricing is likely to have contributed to sales. The Zacks Consensus Estimate for the Animal Health unit is $1.61 billion, while our estimate is $1.58 billion. Nonetheless, a single quarter's results are not important for long-term investors. Let's delve deeper to understand whether to buy, sell or hold Merck stock. Merck's stock has declined 21.1% so far this year compared with a decrease of 2.4% for the industry. The stock has also underperformed the sector and the S&P 500 Index, as seen in the chart below. Image Source: Zacks Investment Research From a valuation standpoint, Merck appears attractive relative to the industry. Going by the price/earnings ratio, the company's shares currently trade at 8.44 forward earnings, lower than 15.07 for the industry as well as its 5-year mean of 13.10. Merck's stock is also cheaper than most other large drugmakers like AbbVie, AstraZeneca, Novo Nordisk and Eli Lilly (LLY). Image Source: Zacks Investment Research Merck boasts more than six blockbuster drugs in its portfolio, with the blockbuster PD-L1 inhibitor Keytruda being the key top-line driver. Merck's animal health and vaccine products are core growth drivers. It has a strong cancer pipeline, including Keytruda. Merck made meaningful regulatory and clinical progress in 2024 across areas like oncology (mainly Keytruda), vaccines and infectious diseases while executing strategic business moves like the acquisitions of Eyebiotech Limited and Harpoon Therapeutics. However, Merck is heavily reliant on Keytruda. Though Keytruda may be Merck's biggest strength and a solid reason to own the stock, it can also be argued that the company is excessively dependent on the drug and should look for ways to diversify its product lineup. There are rising concerns about the firm's ability to grow its non-oncology business ahead of the upcoming loss of exclusivity of Keytruda in 2028. Also, competitive pressure might increase for Keytruda in the near future. In 2024, Summit Therapeutics SMMT reported positive data from a phase III study (conducted in China by partner Akeso) in patients with locally advanced or metastatic NSCLC, in which its lead pipeline candidate, ivonescimab, a dual PD-1 and VEGF inhibitor, outperformed Keytruda. Summit believes iivonescimab has the potential to replace Keytruda as the next standard of care across multiple NSCLC settings. The company's second-largest product, Gardasil, is also seeing grim sales in China. Merck is also seeing weakness in the diabetes franchise and the generic erosion of some drugs. Merck has one of the world's best-selling drugs in its portfolio, generating billions of dollars in revenues. Though Keytruda will lose patent exclusivity in 2028, its sales are expected to remain strong until then. However, the company's problems are too many at present, including persistent challenges for Gardasil in China, potential competition for Keytruda and a bearish outlook for 2025. All these factors have raised doubts about Merck's ability to navigate the Keytruda loss of exclusivity period successfully. No matter how the first-quarter results play out, we suggest that investors with a long-term horizon should stay invested as Merck has strong fundamentals. However, short-term investors should consider selling the stock as the company may take some time to show strong earnings growth. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report AstraZeneca PLC (AZN) : Free Stock Analysis Report Merck & Co., Inc. (MRK) : Free Stock Analysis Report Summit Therapeutics PLC (SMMT) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research Sign in to access your portfolio
Yahoo
12-02-2025
- Health
- Yahoo
FDA approves Gomekli as first neurofibromatosis drug for adults
The US Food and Drug Administration (FDA) has approved SpringWorks Therapeutics' Gomekli (mirdametinib) to treat neurofibromatosis type 1 (NF1), a type of rare genetic disorder. Both adult and paediatric patients with NF1 who have symptomatic plexiform neurofibromas (PN) that cannot be surgically removed will be eligible for the treatment. NF1 is a genetic disorder that currently affects approximately 100,000 children and adults in the US. The NF1 gene codes for neurofibromin, a protein that acts as a tumour suppressor. Patients with the genetic disorder have a 30%-50% lifetime risk of developing PN, which are tumours that grow along the peripheral nerve sheath and are difficult to surgically remove. They can cause skin disfigurement, pain and functional impairment. AstraZeneca's Koselugo (selumetinib) became the first drug approved for NF1-PN in the US in 2020, although it is only indicated for paediatric patients aged two years and older. SpringWorks estimates that around 40,000 people in the US, both children and adults, live with NF1-PN. Both Gomekli and Koselugo are kinase inhibitors that target mitogen-activated protein kinase kinases 1 and 2. These enzymes are responsible for cell growth and are overactive in patients with NF1. SpringWorks' drug demonstrated objective response rates of tumour volume reduction in 41% and 52% of adults and children respectively in the Phase 2b ReNeu trial (NCT03962543). Among those who responded to the treatment, 88% of adults and 90% of children had sustained positive effects after a year. Gomekli's safety and tolerability profile is described as 'manageable', with the most common adverse events across adults and children being vomiting, rash, and diarrhoea. Lead trial investigator Dr Christopher Moertel said: 'It was very encouraging in the ReNeu trial to see that Gomekli provided deep and durable responses, with a manageable safety profile that enabled patients to stay on therapy. This approval represents an important advance, especially for adults who previously did not have an approved treatment.' Gomekli is available in capsules or oral suspension and Connecticut-based SpringWorks expects to distribute the treatment in the US within two weeks. The drug is also currently under regulatory review in Europe, with a decision expected later this year. Due to Gomekli's rare paediatric designation, SpringWorks received a priority review voucher upon approval. This voucher allows the company to fast-track a future candidate of its choice through FDA review, meaning a market placement of four months sooner than usual. SpringWorks could also sell the asset for a quicker cash injection – sales of vouchers have reached more than $100m in recent years. The FDA approval comes at a time when German drugmaker Merck KGgA is eyeing SpringWorks as a potential acquisition. Originally reported by Reuters, Merck confirmed it is in advanced discussions with the Gomekli developer though 'no legally binding agreement has been entered into'. If an agreement is reached, the takeover could happen within a few weeks. SpringWorks share price soared by 34% following the disclosure, bringing its market cap to exceed $4bn. "FDA approves Gomekli as first neurofibromatosis drug for adults" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
