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Eyes on BioPharma News
Jana Chisholm- May 14
- 14 min read
We've been keeping Eyes On BioPharma news, both in the US and Europe. We're sharing a few of the updates from the past few weeks on approval timings, staffing changes, clinical results, international company coalitions, and more.
The FDA has officially confirmed Marty Makary, M.D., as its commissioner. Makary agreed with HHS Secretary Robert F. Kennedy Jr. shortly after a swearing-in ceremony that Peter Marks, M.D., PhD., who had been head of the Centre for Biologics Evaluation and Research for almost ten years, would be replaced by the new administration. Given the option of stepping aside or being pushed away, Marks submitted his letter of resignation.
Kennedy and his anti-vaccine supporters have singled out Marks for criticism and his stance on vaccines at CBER. In his letter of resignation, Marks stated that he was prepared to listen to the public and implement a range of public meetings and engagements with the National Academy of Sciences, Engineering, and Medicine in order to address the Secretary's concerns regarding vaccine safety and transparency.
Marks gained notoriety during his time as a supporter of the FDA's expedited rare disease approval process, particularly when combined with gene treatments. Additionally, he played a significant role in the 2020 Operation Warp Speed initiative, which saw the biopharmaceutical industry and the federal government work together to speed up the development of COVID-19 vaccinations at the height of the pandemic.
It has become evident, according to Marks, that the Secretary wants submissive confirmation of his lies and false information rather than truth and transparency. Although his profile on the FDA's website has been removed, the former CBER officer indicated that he will serve until the first week of April. Under the Trump administration, his departure is one of thousands occurring at federal health agencies. As part of a larger effort to eliminate 10,000 jobs at the Department of Health and Human Services, the Trump administration recently revealed intentions to lay off 3,500 full-time FDA employees and an additional 1,200 NIH employees.
The biopharma sector reacted negatively to Marks's abrupt departure, raising concerns that the loss of competent FDA leadership would undermine scientific standards and have a wider negative impact on the creation of novel, ground-breaking treatments and significantly affect smaller and midsized businesses engaged in gene editing, vaccinations, and cell and gene therapy.
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The FDA has failed to meet yet another approval decision date. Following extensive reorganization efforts throughout the Department of Health and Human Services, the May 7th deadline has passed without a response from the U.S. regulator, despite GSK’s expectations that its IL-5 antibody Nucala would receive an FDA okay for treating chronic obstructive pulmonary disease (COPD).
Getting the FDA to approve Nucala’s sixth COPD nod remains GSK’s goal. In the event that Nucala receives FDA approval for COPD, the biologic will compete directly with Dupixent, the immunological mainstay from Sanofi and Regeneron, which received approval for the condition in September.
The FDA has missed several decision target dates this year. The agency failed to comment on a full approval of Novavax’s protein-based COVID-19 vaccine by the deadline, which was set for early last month. Novavax acknowledged that the FDA had missed its deadline, stating that the company felt its injection was ready for clearance and had answered all of the agency’s information demands.
The FDA has recently postponed approval of elamipretide, a candidate from Stealth Biotherapeutics, for the extremely rare genetic condition Barth syndrome. The FDA informed Stealth in late April that it would not make its deadline and did not provide a new target date for the decision. The HHS stated that in order to maintain the integrity of the review process, the FDA is not permitted to comment on any outstanding applications, and the recent reorganization has nothing to do with these delays.
The FDA pledges to complete standard or priority medication reviews within ten or six months, respectively, under the Prescription medication User Fee Act of 1992. Fees paid under the program by companies seeking FDA approval account for a large amount of the FDA’s overall budget. The user fee collection program helps the industry hold the FDA accountable for conducting timely and effective evaluations, while also providing the agency with the necessary resources to fulfill its responsibilities.
The recent elimination of thousands of jobs at HHS, including 3,500 full-time FDA employees, under Secretary Robert F. Kennedy Jr. has sparked serious concerns about the regulator’s capacity to perform essential duties like inspections and timely reviews, even though it is difficult to pinpoint exactly what is going on behind the FDA’s doors at the moment. According to the former director of the FDA’s Centre for Biologics Evaluation and Research, who resigned in March, the organization now faces the significant danger of struggling to adhere to its medication review schedules in the coming year or two. However, FDA employees remain dedicated to conducting timely reviews, the former director emphasized. The adjustments that have been made public are only the beginning of the significant changes that have taken place at the organization.
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Amgen, Astra Zeneca, Biogen, Bristol Myers Squibb, Johnson & Johnson, Novartis, Pfizer, Roche, Sanofi, and other multinational pharmaceutical companies are urging the UK government to amend a drug cost control law to give the industry greater leeway for expansion.
The British government initially imposed a ceiling on the increase of branded medicine spending by the National Health Service in 2014, which has been criticized by the pharmaceutical sector. A prior agreement between the UK government and the pharmaceutical sector stated that the NHS may spend 2% more on branded medications annually in 2024 and 4% more by 2028.
With a mechanism to make modifications annually rather than every five years based on revisions to annual NHS budgets, pharmaceutical companies now want the permissible growth rate to reflect improvements in overall NHS funding. Additionally, biopharma firms have suggested implementing "risk-share mechanisms" that would increase the UK government's accountability for covering rising pharmaceutical costs.
Due to the spending growth cap, the UK government declared in December 2024 that, as part of a drug cost control policy called the voluntary scheme, or VPAG, companies would have to pay a 22.9% rebate on sales of newer medications in 2025, in addition to an additional 0.6% contribution to an investment program. A prior estimate of 15.3%, derived from historical sales data, was significantly exceeded by the current figure.
The significant rise is pushing the voluntary program to its breaking point. Businesses have stated unequivocally that current prices render the UK uninvestable from a global standpoint and will harm the introduction of new medications if they remain the same. This will put the government's goals for the upcoming NHS 10-Year Health Plan and Life Sciences Sector Plan in jeopardy.
In 2023, biopharma companies paid out £2.5 billion ($3.2 billion) in rebates under the old program, which accounted for 21.2% of the NHS's overall spending on branded medicine sales. Pharmaceutical companies want the freedom to review the overall cost growth cap figure in combination with annual NHS budgets when the UK government increases the rebate rate based on more recent sales data. Medicines should get the same proportionate funding boost as the rest of the NHS, according to the industry's proposal.
Due to a general NHS budgetary limitation during the previous UK administration, the industry agreed on the cap levels in the 2024–2028 contract. But since then, the present administration has significantly increased financing for the UK health system, which has increased the use of medications. In addition to imposing a strict cap on the growth of medication spending, the industry is putting up "risk-share options" that might involve distributing growth gains above certain thresholds equally or modifying permitted growth levels to rise in tandem with market expansion.
Taking inflation into account, the UK's branded medication industry has actually decreased by 11% over the last decade, largely due to the spending cap. In contrast, the NHS funding has increased by 33% during the same time frame. Approximately 9% of the UK's overall health spending goes towards medicines, which is less than that of other developed countries like the US (14%), Germany (17%), and Japan (17%).
With fewer clinical trials, fewer new drugs being introduced, fewer collaborations supporting the NHS, and personnel reductions throughout the business, the UK life sciences sector is in decline. Ahead of the VPAG's scheduled review this autumn, the Association of the British Pharmaceutical Industry is now urging for in-depth discussions with the government. If not, businesses might leave the program, which would cause it to fail. This would have a significant impact on how stakeholders worldwide view the UK and would also increase the risk to the Life Sciences Sector Plan's reception by investors.
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GSK's neuroscience strategy has been further developed with a $2 billion ($2.5 billion) agreement to use a South Korean company's technology to get over the blood-brain barrier. To assist the delivery of medications for neurodegenerative diseases across the infamously difficult barrier and into the brain, ABL Bio created the Grabody-B platform, which targets the insulin-like growth factor 1 receptor (IGF1R). GSK has agreed to acquire Grabody-B-related technology and expertise from the Seongnam South Korea-based biotech. The British Big Pharma will be responsible for developing any ensuing medications, from preclinical research to commercialization.
GSK will pay £ 38.5 million ($49.5 million) upfront, with an additional £ 38.6 million ($49.6 million) potentially due in the near future. The deal's total biobucks rise to 2.07 billion pounds ($2.5 billion) when longer-term research, development, and commercialization payments are also considered, along with GSK's potential to broaden the program's scope.
By signing an experimental agreement with Danish startup Muna Therapeutics to study postmodern human brains in an attempt to find multiple new Alzheimer's disease treatment targets, GSK broadened its neuroscience expertise at the end of last year. This was in addition to a deal with Vesalius Therapeutics, created by Flagship, that featured a preclinical small molecule that was first intended to treat Parkinson's disease. Sanofi and I-Mab, for example, have already leveraged ABL to develop bispecific antibodies for gastric cancer and Parkinson's disease, respectively.
The biotech views the deal with GSK as an ideal opportunity to increase the various areas where Grabody-B can be used and to solidify ABL Bio's position in the neurodegenerative disease therapy market through the possible commercialization of Grabody-B.
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The Vaccine Integrity Project, armed with $240,000, is starting to protect vaccination usage in the face of extensive disinformation campaigns and federal funding cuts to public health initiatives. With funding support from iAlumbra, the program was launched last week by the Centre for Infectious Disease Research and Policy (CIDRAP) at the University of Minnesota. An eight-member steering committee of public health and policy professionals from the United States will lead the program and offer recommendations for sustaining vaccination usage that are grounded in the most up-to-date research.
Margaret Hamburg, M.D., a former FDA commissioner and current co-president of the InterAcademy Partnership, a worldwide partnership for academies of science, medicine, and engineering, will serve as co-chair of the new committee. Harvey Fineberg, M.D., PhD, a former president of the Gordon and Betty Moore Foundation, is the other co-chair. The group will provide vaccine-related recommendations to nongovernmental organizations, including CIDRAP, that are independent and dedicated to defending Americans against diseases that can be prevented by vaccination.
The Vaccine Integrity Project will hold information-gathering sessions over the course of the next four months with state health officials, medical associations, public health organizations, vaccine producers, academic experts, insurers, healthcare systems, pharmacists, and legislators. The project's scope, membership requirements, and operational aspects will be influenced by those conversations.
The group attributed the launch to an increasing number of voices raising doubts about the effectiveness and safety of vaccines as well as a growing mistrust among Americans. Examples include orders from Robert F. Kennedy Jr., secretary of the Department of Health and Human Services. Under his direction, research on the already debunked theory that vaccines cause autism has been started by the Centres for Disease Control and Prevention.
In addition, RFK Jr. drastically reduced the government health agency's research, activities, and workforce. Earlier this month, RFK Jr. approved the measles, mumps, and rubella vaccination in response to a measles outbreak in the United States. The CIDRAP also referenced recently hired Jayanta Bhattacharya, M.D., Ph.D., Director of the National Institutes of Health, who stated in a July 2024 post on the social media platform X that he would be in favor of rescinding regulatory approval for COVID-19 mRNA vaccines.
The institution cited a bill presented last week by eight Republicans in the Minnesota House of Representatives that would designate mRNA vaccinations against COVID-19 as weapons of mass destruction. The plan would make obtaining or using the therapy illegal and carry a maximum 20-year prison sentence.
The Advisory Committee on Immunisation Practices, which advises the CDC on vaccinations, is not replaced by the new program, according to CIDRAP.
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Novo Nordisk, a competitor of Eli Lilly in the metabolic medicine market, has secured a new formulary access agreement that may provide its flagship weight reduction medication, Wegovy, with a significant edge over its Lilly counterpart, just as Eli Lilly seemed to be gaining ground on Novo in the U.S. obesity market. Wegovy will be the recommended GLP-1 for treating obesity on CVS Caremark’s largest commercial template formularies as of July 1st. In the United States, CVS Caremark is the biggest pharmacy benefit manager. It is part of the prescription benefits division of the chain of pharmacies of the same name.
With a CVS confirming that Lilly’s medication will be removed in health plans utilizing CVS’ standard list of covered pharmaceuticals, the action will remove Zepbound, Lilly’s dual GIP/GLP-1 receptor agonist for obesity, off CVS Caremark’s current formulary. Individuals currently using Zepbound on these programs will have the option to switch to Wegovy. On an individual basis, they may also be eligible for exemptions due to medical necessity.
The formulary modification will essentially increase access to Wegovy in the United States, likely at the expense of Lilly’s Zepbound. The CVS agreement is part of Novo’s continuous endeavor to increase patient access to Wegovy, which has been difficult for patients to obtain in recent years due to supply issues, a high cost, and insurers’ reluctance to cover the enormously popular GLP-1 medicine class.
The pharmaceutical company introduced NovoCare Pharmacy in March, which provides direct home delivery for a discounted monthly fee of $499. That pricing is much less than the current list price of $1,349 per package and covers all five of the medication’s dose levels. The firm recently modified its savings plan to allow patients who pay out of pocket to also receive the $499 monthly pricing at nearby physical pharmacies.
With the new agreement, Novo is allowing the pharmacy giant to offer Wegovy to cash-paying consumers at its more than 9,000 locations across the United States for $499.
The deal is a setback for Novo’s rival Eli Lilly, but the CEO of the company was not shocked by the announcement, acknowledging that the private pay market is significant and that he wants Lilly to expand in that space while simultaneously giving out-of-pocket customers more options. He emphasized that his organization has no interest in one-on-one agreements that limit patients’ and doctors’ access and options.
When Lilly announced that it would lower the prices of its 2.5-mg single-dose Zepbound vials to $349 per month and its 5-mg vials to $499, it was emulating Novo’s out-of-pocket discount for Wegovy in late February. The company claimed that it was figuring out the move because insurance and federal programs do not routinely cover medical care for individuals with obesity in the United States.
In the meantime, Wegovy’s dealmaking table with Novo was not just for CVS. The company said that it is utilizing its NovoCare online pharmacy to help patients obtain branded Wegovy by partnering with three telehealth companies: Hims & Hers, LifeMD, and Ro.
A $599 packaged monthly membership that includes prescriptions for all dosages of Wegovy, access to round-the-clock care, continuing clinical assistance, and dietary advice is being offered by Hims & Hers as part of the packages. Wegovy will be available for $499 a month from Ro and LifeMD, along with other weight-loss programs.
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Pfizer discontinued its oral GLP-1 product, danuglipron, following a phase 1 trial in which one patient may have suffered liver damage as a result of the medication. The failure puts a dent in the Big Pharma’s strategy to compete in the obesity industry.
Two phase 1 dose-optimization studies were conducted by the business to investigate once-daily danuglipron formulations. Pfizer reported that the studies achieved important pharmacokinetic goals and validated a dosage and formulation that potentially provide phase 3 tolerability and competitive efficacy. However, the trials also ended the development of the GLP-1 receptor agonist.
Following the discovery of possible drug-induced liver damage in one patient, Pfizer decided to discontinue operations. After the patient stopped taking danuglipron, the injury went away. According to Pfizer, the overall prevalence of elevated liver enzymes among the more than 1,400 patients in the safety database was consistent with that of the class’s approved medications.
Nevertheless, Pfizer was convinced to axe the asset after reviewing its clinical data and receiving feedback from authorities. Given the degree of competition in the oral GLP-1 market, patients and doctors may have access to therapies with a cleaner safety profile if competitors steer clear of the liver toxicity pitfalls that Pfizer encountered.
In 2023, Pfizer discontinued lotiglipron, another oral GLP-1 candidate, due to increased liver enzymes observed during clinical testing. Later that year, the Big Pharma chose to continue developing a once-daily formulation of danuglipron instead of a twice-daily one, which might have presented a greater threat to competitors like Eli Lilly, Structure Therapeutics, Viking Therapeutics, Astra Zeneca and Novo Nordisk. Danuglipron’s discontinuance eliminates the main component of Pfizers’s obesity campaign and makes one wonder whether the business will reach an agreement to regain its position in the competition.
For adults with obesity, Pfizer is developing a phase 2 oral small molecule GIPR antagonist that, according to the company, may be used in a fixed-dose combination with danuglipron. For the time being, a GIPR-GLP-1 combo is no longer a possibility as a result of the withdrawal.
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Alnylam hopes that its RNA interference strategy will help it stand out from its rivals in the transthyretin amyloid cardiomyopathy (ATTR-CM) arena, which is becoming increasingly competitive. In support of this, the pharmaceutical company just received a new FDA approval.
For people with wild-type or inherited ATTR-CM cardiomyopathy, the FDA has approved Alnylam's Amvuttra, also referred to as vutrisiran, as a medication to lower the risk of cardiovascular death, hospitalizations, and urgent heart failure visits.
Alnylam stated in a recent statement that Amvuttra is now the first drug authorized in the United States to treat both ATTR-CM and polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN), following a prior approval in 2022.
Pfizer, whose Vyndaqel (tafamidis) family of transthyretin (TTR) stabiliser medications received their first ATTR-CM nods from the FDA in 2019, now dominates a potentially profitable market that Amvuttra is entering. In the meantime, BridgeBio's TTR stabilizer, Attruby, obtained FDA approval in November, and its launch has been going well so far. However, as TTR stabilizers have been the only medications available for the disease thus far, the approval of a new method of action will broaden the scope of this severe but mostly underdiagnosed condition.
In its approval statement, Alnylam stated that Amvuttra is an RNA interference medication that helps target ATTR-CM at its source by quickly knocking down TTR. By rapidly inhibiting TTR production, Amvuttra reduces the accumulation of TTR fibrils, which create amyloid and result in irreversible cardiovascular damage and early mortality in ATTR-CM patients.
Alnylam's phase 3 Helios-B study met all 10 of the FDA's primary and secondary objectives, and the FDA used this data to support its clearance decision. During a double-blind treatment term of up to 36 months, Amvuttra showed a 28% reduction in the risk of all-cause death and recurrent cardiovascular events compared to placebo in the entire group of patients. During the trial's open-label extension phase, the same Amvuttra study sample saw a 36% decrease in mortality over 42 months.
Approximately 170 healthcare systems provide about 80% of all ATTR-CM prescriptions in the United States, and approximately 3,700 physicians treat 90% of ATTR-CM patients. Alnylam used to get in touch with between one-third and forty percent of those cardiology centers. The company's current goal is to attain 100% in order to cast a wide net.
The annual cost of Amvuttra in cardiomyopathy, before discounts, is approximately $450,000. Although that price is comparable to the drug's cost in polyneuropathy, it is significantly higher than that of BridgeBio's Attruby in ATTR-CM and Pfizer's Vyndaqel products. As income from the cardiomyopathy indication begins to accumulate, Amvuttra's net price may eventually decrease due to rebates and other concessions, according to Alnylam.
In its official statement, the organization also mentioned that 99% of patients in hATTR-PN have insurance coverage for Amvuttra, with the majority paying nothing out of pocket for the medication. According to Alnylam, it anticipates achieving comparable out-of-pocket expenses and extensive coverage in the cardiomyopathy indication.
Alnylam has a chance to make a significant impact on sales with the new approval. Pfizer announced Vyndaqel sales of over $5.4 billion last year, a 64% increase from the previous year, to give a sense of the potential possibility at stake.
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