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The US Centers for Medicare & Medicaid Services' power to negotiate certain drug prices under the Inflation Reduction Act (IRA) has been criticised by the pharmaceutical industry for myriad reasons. While drugmakers' attempts to overturn the programme as a whole have been repeatedly rejected, a legislative fix was in the works to alleviate a specific pain point concerning orphan drugs — but the bill's chances of making it into law are now looking slim.Under the IRA, drugs that have been on the market for 7 years — or 11 for biologics — are eligible for pricing negotiations. While therapeutics approved by the FDA for a single orphan indication were exempt, if the treatment was later cleared for a second rare disease, or a non-orphan indication, it could then be subject to pricing talks — with its on-market period calculated from its first orphan approval.The ORPHAN Cures Act, introduced by Congressmen Don Davis and John Joyce into the US House of Representatives in February, aimed to expand the orphan drug exclusion so that therapeutics approved for multiple rare diseases would not be eligible for negotiations. Additionally, if an orphan drug subsequently was greenlit for a non-orphan indication, the timeline to determine its eligibility for pricing talks would be based on that second, non-rare disease approval. The Biotechnology Innovation Organization (BIO) trade group threw its support behind the ORPHAN Cures Act, saying that it would "ensure the continuation of essential research and development of new medicines for patients with rare diseases." BIO criticised the IRA's current orphan drug provisions, claiming they "disincentivize companies to test whether existing treatments for rare diseases also benefit other rare diseases."The ORPHAN Cures Act seemed to be making positive headway after it was included in the House's 2025 budget, which was approved in May. However, it appears to have been excluded from the Senate's version of the bill, per reconciliation text released by the finance committee this week. While the future of the ORPHAN Cures Act is uncertain, another piece of the IRA that has been widely criticised by industry — the so-called "pill penalty" discrepancy between when drugs and biologics are eligible for pricing negotiations — is looking more likely to be fixed. President Donald Trump signed an executive order in April directing Congress "to align the treatment of small molecule prescription drugs with that of biological products, ending the distortion that undermines relative investment in small molecule prescription drugs."

Eli Lilly has already secured several billion-dollar deals in 2025. Credit: JHVEPhoto via Shutterstock. Eli Lilly has agreed to acquire cardiovascular disease treatment specialist Verve Therapeutics in a deal potentially rising to $1.3bn, with market analysis describing the deal as a “bargain”. According to the transaction, Eli Lilly will purchase all Verve shares for $10.50 each, with the drugmaker outlaying an additional $3 in contingent value right (CVR) per share. Verve’s pipeline includes gene editing medicines that are “designed to address the drivers of atherosclerotic cardiovascular disease”. Similar to other advanced therapies on the market for certain diseases, the biotech is targeting one-time administration. The total potential Eli Lilly deal value represents a 115% premium to Verve’s share price at market close yesterday (16 Jun). Shares in Nasdaq-listed Verve surged to $11.03 at market open on 17 June following the announcement, a 43% increase on the previous market close. William Blair analyst Myles Minter said in a research note: “Ultimately, we believe that the deal makes sense for Verve shareholders and makes sense given the exposure Lilly has to Verve’s entire disclosed pipeline. Although we believe Eli Lilly is getting a bargain here (our fair value was $30.86 per share prior), the 115% premium (assuming full CVR payout) is still a win for Verve shareholders and the gene editing space more broadly, which has been under significant macro pressure in a difficult funding environment.” Already in partnership with Eli Lilly, Verve recently reported positive Phase Ib data for its lead programme VERVE-102, a therapy targeting PCSK9, a gene connected to cholesterol levels and cardiovascular health. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence The Heart-2 study demonstrated that VERVE-102 exceeded the cholesterol reduction achieved by Novartis’ Leqvio (inclisiran), an injectable drug that dominates the market. Apart from the second dose, which is three months apart, Leqvio is dosed every six months. In his analysis, Minter added: “We remain impressed by the Heart-2 dataset for VERVE-102 and believe Lilly likely wanted greater control over later-stage clinical development.” Eli Lilly has already spent big in 2025. The drugmaker purchased a cancer asset from biotech Scorpion Therapeutics for up to $2.5bn , and agreed to acquire pain treatment specialist SiteOne Therapeutics for up to $1bn. June spending goes big for big pharma Despite an uncertain economic landscape in the US, courtesy of President Donald Trump, mergers and acquisitions (M&A) activity has ramped up in 2025. Total deal value in Q1 surged 101% compared to Q4 2024, as per analysis by GlobalData . Pharma companies rallied to spend a total of $37.7bn. This week alone has already seen deals surpass the billion-dollar mark. BioNTech, for example, agreed to acquire mRNA specialist CureVac for $1.25bn earlier this week. Elsewhere in the partnership arena, AstraZeneca outlaid $5.2bn to enlist the services of China-based CSPC Pharmaceuticals. In addition, BMS earmarked $11bn earlier this month to join BioNTech’s development of a bispecific cancer drug. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. 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iStock, Deagreez Big Pharma executives have not been shy about their desire for deals, but companies have been battling macro headwinds alongside Trump’s policies on drug pricing and tariff threats. A string of deals has analysts optimistic for more activity in the second half, as total deal value for the first half is nearing the sum of all 2024 deals. Pfizer, Biogen, Merck, Eli Lilly and Gilead still have capital to deploy, according to Jefferies, and there are plenty of biotechs that might fit neatly into some cracks in these Big Pharmas’ pipelines. First, Sanofi made the second biggest acquisition of the year so far with $9.5 billion offered for rare disease biopharma Blueprint Medicines . Then BioNTech pledged $1.25 billion to buy rival CureVac , which got BMO Capital Markets wondering if the dam was finally breaking and momentum beginning to build. And on Monday, Sage Therapeutics was acquired by Supernus Pharmaceuticals for up to $795 million. “It feels as if we are starting to get some traction in the sector with some recent ASCO deals ( Blueprint Medicines ) and now CureVac. Still, macro concerns remain, but we appreciate recent developments in M&A,” BMO’s Evan Seigerman wrote on Thursday. Jefferies also noted Intra-Cellular, which was acquired by Johnson & Johnson for $14 billion to kick off the year. Thanks to these larger acquisitions, total deal value for this year has already reached $36 billion, compared to $44 billion for all of 2024. Big Pharma executives have not been shy about their desire for deals, but companies headed into the year still battling macro headwinds and added overhangs of the Trump administration moves on drug pricing and tariffs. “From a macro perspective, uncertainty from potential sector specific tariffs and new drug pricing policies make investment decisions more challenging for BD teams but we think we have passed ‘peak negativity,’” Jefferies said in a Friday report. Even if tariffs result from the Trump administration’s Section 232 review, Jefferies thinks they will be “less bad than feared.” Biopharma stocks have been volatile along with the broader markets as policies are proposed and retracted. Jefferies said that this kind of environment does not typically breed deals as companies wait for clarity but sees the markets and valuations stabilizing over the next three to five months. TThe FDA has been a source of uncertainty, after failing to meet several PDUFA deadlines in recent weeks, while simultaneously meeting or even beating other approval decision timelines. That had been a major concern after the agency gutted its workforce after President Donald Trump took office. Which Biotechs Will Get Bought? If the S&P Biotechnology Select Industry Index, the XBI, does stabilize, companies could begin scrambling for deals after a long drought. Jefferies has some ideas of where we might see deals. Akero Therapeutics is a “highly de-risked attractive M&A candidate,” according to Jefferies. The biotech could have potential sales of $2–3 billion and has strong Phase II data for a therapy in cirrhosis metabolic dysfunction-associated steatohepatitis (MASH). This space has recently been reinvigorated after GSK bought Boston Pharma for $2 billion in May . The space had seemed relatively closed after the May 2024 approval of Madrigal Pharmaceuticals’ Rezdiffra, but now it seems Big Pharma is again interested. Another potential target Jefferies identified is Scholar Rock , which is expecting an FDA decision for apitegromab in spinal muscular atrophy in September. This would provide a lucky buyer with a near term revenue-generating asset—in addition to a Phase II obesity asset, with for that drug due this month. Obesity in general could be a major target for Big Pharma, with the leaders in that space — Novo Nordisk and Eli Lilly —signing a flurry of licensing deals of late. More dealmaking in China , which has been sprouting myriad programs for U.S.-based companies to license, could also be on the horizon. This could unfortunately come at the expense of U.S.-based biotechs, Jefferies noted. “Pharma/large-cap companies have benefited from in-licensing Chinese assets at attractive valuations, but this makes for a tougher M&A environment for U.S. small-caps as pharma increasingly turns to overseas business development opportunities instead of shopping local,” Jefferies said.