Calliditas Therapeutics AB won full approval from the U.S. FDA for Tarpeyo (budesonide) delayed release capsules to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression. Shares of the company (NASDAQ:CALT) were trading at $25.74, up $3.64, or 16%. Given the FDA go-ahead two years ago under the accelerated approval pathway by way of data based on the surrogate marker of proteinuria, Tarpeyo with the latest regulatory advance has become the first fully FDA-approved treatment for IgAN as measured by kidney function.

Merck’s gefapixant gets second CRL for chronic cough

Receiving a second complete response letter (CRL) from the U.S. FDA for gefapixant to treat refractory and unexplained chronic cough – an indication for which there are no approved treatments in the country, Merck & Co. Inc. said it is reviewing the agency’s feedback to determine the next steps, if any, for the oral selective P2X3 receptor antagonist. The CRL came as no surprise to investors as the U.S. FDA’s Pulmonary-Allergy Drugs Advisory Committee voted 12-to-1 against the drug in November. 

Top Trends of 2023: European firms lament ‘tax on growth’ as rebate inflation hits budgets

Pharma companies facing the pricing pressures unleashed by the U.S. Inflation Reduction Act will find little respite in European markets in 2024, as governments erect higher market access hurdles around pricing and reimbursement in a bid to constrain drug budgets. Added to this are rebate schemes that require companies to pay back a percentage of their revenues if the total national bill goes up too much. From the industry’s perspective, these controls do not recognize the value of innovation. For the payers, rebates are central to their ability to afford products, such as cell and gene therapies and orphan medicines, that have huge price tags.

Final guidances pouring out of US FDA

With the clock ticking down on 2023, the U.S. FDA seems to be scurrying to push final guidances out the door. This week, the agency has finalized several guidances, ranging from the development of monoclonal antibodies to treat COVID-19 infections to the use of real-world data in drug development. The agency also issued a final rule allowing institutional review boards to waive or alter informed consent for certain investigations.

Top Trends of 2023: Psychedelic-assisted therapy: The state of play in Europe

Following the decision of Australia’s Therapeutic Drugs Administration to allow prescribing of MDMA for post-traumatic stress disorder and psilocybin in treatment-resistant depression from July 2023, and with U.S. FDA approval of MDMA for treating PTSD expected in 2024, the EMA is under increasing pressure to set out a path to approval for psychedelics. In February 2023, six members of the European Parliament wrote an open letter to Emer Cooke, executive director of the EMA, pointing to these and other regulatory advances in the U.S. and Australia, and to an increasing body of evidence that psychedelic-assisted psychotherapy can be a highly effective treatment for mental health disorders. 

Pinotbio, Conjugatebio strike $250M deal for 10 ADC targets

South Korean biotech Pinotbio Co. Ltd. inked a $250 million (₩320 billion) licensing deal with Princeton, N.J.-based Conjugatebio Inc. to develop 10 antibody-drug conjugate (ADC) targets with Pinotbio’s ADC platform technology. The deal announced on Dec. 21 adds to a previous agreement between the two companies last year. Pinotbio and Conjugate first teamed up to discover and develop five ADC targets in June 2022. Combined, the two firms will develop up to 15 ADC targets with Pinotbio’s camptothecin-based ADC linker-payload platform dubbed the Pinot-ADC.  

Top Trends of 2023: U.S. FTC probes rattle biopharma, but deals still getting done

A threat to biopharma innovation arrived at a larger scale in 2023 in the form of greater U.S. FTC scrutiny, calling into question the legitimacy of certain M&As and deals. At top of mind throughout part of the year was whether Amgen Inc.’s $27.8 billion offer in December 2022 to buy Horizon Therapeutics plc would culminate into a closed deal, after the commission – in an unprecedented action – challenged the merger, not because of overlapping products, but because of concerns that Thousand Oaks, Calif.-based Amgen would bundle its high-volume products with two of Dublin-based Horizon’s orphan drugs when dealing with pharmacy benefit managers. The deal closed in November after Amgen formally agreed not to bundle the products. 

Some normalcy returning to US FDA with more in-person meetings

Nearly four years after the COVID-19 pandemic closed government offices and sent most federal employees home to work remotely, the U.S. FDA is returning to some semblance of normalcy, with its drug and biologics centers expanding in-person face-to-face industry meetings to include all PDUFA and BsUFA meeting types, beginning Jan. 22. However, the in-person meetings won’t be automatic, as a sponsor must specifically request one. Then the FDA’s review division will determine whether to grant the request. Even if one is granted, it still will have a virtual component.

BioWorld Insider Podcast: Six biopharma executives consider a tough 2023 and are hopeful for a better 2024

One aspect of 2023 our group of executives completely agreed on: the past year was tough financially. And they all foresee a more vibrant year ahead for the market. Giving all of them hope were technological breakthroughs such as artificial intelligence, game changing weight loss drugs, the surging fascination with ADCs and hope for lower interest rates. But uncertainty looms about the upcoming general election in the U.S. and ground-shifting gene therapies. In a preview of the annual Biotech Showcase conference, an investor conference for private and micro- to mid-cap biotech companies Jan. 8-10 in San Francisco, BioWorld spoke with Dave Bearss, CEO of Halia Therapeutics Inc., Vimal Mehta, CEO of Bioxcel Therapeutics Inc., Paul Lammers, CEO at Triumvira Immunologics Inc., Chris Pirie, COO of HDT Bio Corp., Thijs Spoor, CEO of Perspective Therapeutics Inc., and Shelley Hartman, CEO of Aegle Therapeutics Corp. They offered insights brought about by years of hard-won experience.

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Also in the news

Acelyrin, Alexion, Alterity, Anavex, Annexon, Argenx, Ascendis, Atara, Aurinia, Barinthus, Briacell, Cellvax, Chiesi, Compass Pathways, Cstone, Daré, Disc Medicine, DMK, Edenbridge, Enlivex, Eterna, Exscientia, Galapagos, Gilead, Hervolution, hookipa, Iama, Impact, JCR, Kancera, Lift, Longeveron, Lyndra, Lynk, Meiragtx, Myriad Genetics, Nectin, Neuren, Ocelot, Oculis, Pacira, Panbela, Passage, Pfizer, Pierre Fabre, Pluri, Rallybio, Rnaimmune, Sanofi, Scitech, Shattuck Labs, Takeda, Tme, Tonix, Tracon, Uvax