Sanofi Cablivi approved for rare blood disorder in pediatric patients 12 years and older

 The U.S. Food and Drug Administration approved Cablivi (caplacizumab-yhdp) for injection to treat pediatric patients 12 years and older with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy. Cablivi was originally approved in 2019 to treat adults with this condition. 

Disease or Condition

Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare disorder that is even more uncommon in children, only occurring in about 1 per 10 million children annually. aTTP causes blood clots in the small blood vessels. These clots can slow or block the flow of blood to parts of the heart, brain, kidneys, and other organs. In this disorder, platelets (blood cells involved in clotting) clump together, leaving fewer platelets in the rest of the body to help with clotting. In addition, red blood cells may break apart, causing anemia.

Data Supporting Cablivi

The efficacy of Cablivi to treat pediatric patients with aTTP was evaluated in a retrospective chart review study of 30 patients ranging in age from 2-18 years old. Results showed that 80% of patients achieved clinical remission, defined as achieving a normal platelet count and having LDH levels (a marker of red blood cell breakdown) less than 1.5 times the upper limit of normal for 30 days or more.

Safety Information

Because Cablivi works by targeting the abnormal blood clotting, it can also cause serious and fatal bleeding. The risk of bleeding is higher in patients with underlying coagulopathies (impaired clotting abilities) or who are also taking blood thinning medicines (antiplatelet agents or anticoagulants). Patients should stop treatment if clinically significant bleeding occurs. Patients should also not take Cablivi 7 days before elective surgery, dental procedures, or other invasive procedures. In addition, patients should not take Cablivi if they have had a severe allergic reaction to any of its ingredients.

The most common side effects are nose bleeds, headache, and gingival (gum) bleeding.

Designations

Cablivi received Orphan Drug Designation for this indication.

https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-therapy-rare-blood-disorder-pediatric-patients-12-years-and-older

Incyte's blood cancer therapy slows disease progression in trial

 Phase 3 frontMIND trial evaluating the efficacy and safety of tafasitamab (Monjuvi®/Minjuvi®) and lenalidomide in addition to R-CHOP met its primary endpoint of progression-free survival (PFS) - Based on these results, Incyte plans to file a supplemental Biologics License Application (sBLA) for tafasitamab and lenalidomide in addition to R-CHOP in first-line diffuse large B-cell lymphoma (DLBCL) in the first half of 2026

Incyte (Nasdaq:INCY) today announced positive topline results from the pivotal Phase 3 frontMIND trial evaluating the efficacy and safety of tafasitamab (Monjuvi®/Minjuvi®), a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody, and lenalidomide in addition to R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone) compared to R-CHOP alone as a first-line treatment for adults with newly diagnosed diffuse large B-cell lymphoma (DLBCL) with an International Prognostic Index (IPI) score of three to five (3-5) for patients >60 years of age, or age-adjusted IPI (aaIPI) of two to three (2-3) for patients ≤60 years of age.

The trial met its primary endpoint of progression-free survival (PFS) by investigator assessment (Hazard Ratio 0.75 [0.59,0.96]; p-value 0.019), according to Lugano 2014 criteria. The trial also met its key secondary endpoint of event-free survival (EFS) by investigator assessment. No new safety signals were observed.

“The frontMIND study results highlight the potential benefit of combining tafasitamab and lenalidomide with R-CHOP as an effective treatment option, offering the possibility of cures for more newly diagnosed DLBCL patients,” said Steven Stein, M.D., Chief Medical Officer, Incyte. “Despite improvement in treatment for patients with DLBCL, outcomes for many high-risk patients are not optimal. We look forward to working with regulatory authorities globally and to providing a new treatment option for patients in the future.”

DLBCL is the most common type of non-Hodgkin lymphoma (NHL) in adults worldwide, representing 40% of all cases.1 It is characterized as an aggressive, fast-growing type of lymphoma that can emerge in lymph nodes or extranodal sites such as the gastrointestinal tract, skin and brain.2 Each year, approximately 24,000 people in the U.S. and up to 36,000 people in Europe are diagnosed with DLBCL.3,4,5,6 With about 40% of these patients not responding to initial therapy or relapsing thereafter7,8, there is a high medical need for new, effective therapies.

Based on these positive results, Incyte expects to file a supplemental Biologics License Application (sBLA) for tafasitamab for the first-line treatment of adults with newly diagnosed DLBCL in the first half of 2026. The frontMIND data will be submitted for presentation at an upcoming scientific meeting.

Tafasitamab was approved in combination with lenalidomide by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in 2020 and 2021 respectively, for adult patients with relapsed or refractory DLBCL not otherwise specified including DLBCL arising from low-grade lymphoma, and who are not eligible for autologous stem cell transplant. Additionally, tafasitamab was approved in combination with lenalidomide and rituximab by the FDA in June 2025 for adult patients with relapsed or refractory follicular lymphoma (FL). In November 2025, the EMA’s Committee for Medicinal Products for Human Use issued a positive opinion recommending the approval of tafasitamab for patients with relapsed or refractory FL.

https://www.marketscreener.com/news/incyte-announces-positive-topline-results-from-pivotal-study-of-tafasitamab-monjuvi-minjuvi-as-a-ce7e59dedb80f621

Minneapolis Hilton hotel cancels immigration agents' reservations, DHS says

 

The U.S. Department of Homeland Security on Monday said a Hilton hotel canceled reservations for Immigration and Customs Enforcement agents in Minneapolis, where the Trump administration has surged officers after allegations of fraud against Somali immigrants.

After ICE officers booked rooms using official government emails and rates, Hilton canceled their reservations, DHS said in a post on social media platform X.

The department also posted screenshots of emails, in which the hotel's operators said they had "noticed an influx of GOV reservations made today that have been for DHS," and that they were not allowing any ICE agents to stay at the property.

Hilton Worldwide Holdings said the property is independently owned and operated and is investigating the matter.

"Hilton works with governments, law enforcement and community leaders around the world to ensure our properties are open and inviting to everyone," a spokesperson said in a statement. 

Federal officials in recent weeks have portrayed Minnesota's Somali community as a hotspot for fraud involving millions of federal dollars intended for social services. Immigrant-rights advocates have accused the administration of using the fraud investigations as an excuse to target Somali immigrants more broadly.

Shares of the hotel chain were down 1.5% in afternoon trading.

https://www.marketscreener.com/news/hilton-s-minneapolis-hotel-cancels-reservations-for-law-enforcement-officers-dhs-says-ce7e59dedf88f12d

Atlanta Fed: Q4 GDPNow estimate down to 2.7%

 The Federal Reserve Bank of Atlanta announced on Monday in its GDPNow model prospects that for the fourth quarter of 2025, real GDP growth in the United States is expected to stand at 2.7% as opposed to 3% seen on December 23.

Meanwhile, the nowcast for the real personal consumption expenditures growth in the last quarter of 2025 was revised down from 2.7% to 2.4%, while the projections for the real gross private domestic investment growth declined from 7% to 6.5%.

https://breakingthenews.net/Article/Atlanta-Fed:-Q4-GDPNow-estimate-down-to-2.7/65426333

Bessent: US firms exempt from OECD global tax

 United States Secretary of the Treasury Scott Bessent announced on Monday that the US has finalized an agreement to exempt US multinationals from the Organisation for Economic Co-operation and Development's (OECD) Pillar Two global minimum tax.

Bessent said the deal, reached in coordination with Congress and over 145 countries in the OECD/G20 Inclusive Framework, protects incentives such as the research and development (R&D) tax credit. "This side-by-side agreement recognizes the tax sovereignty of the United States over the worldwide operations of U.S companies and the tax sovereignty of other countries over business activity within their own borders," he concluded.

Treasury said it will continue talks with other countries to support implementation and promote international tax stability.

https://breakingthenews.net/Article/Bessent:-US-firms-exempt-from-OECD-global-tax/65426778

Zelensky to replace security service head

 Ukrainian President Volodymyr Zelensky revealed on Monday that he had met with the Head of the Security Service (SBU), Vasyl Malyuk, thanked him for his service, and discussed his successor.

While omitting to name any individual, Zelensky said that Malyuk "knows how to do this best and will continue to do this in the SBU system," praising his contribution in combat and instructing him to pursue even "stronger results" in operations against Russia aimed at "destroying the enemy."

Malyuk has held the position since February 2023, and before served as Acting Head of the SBU from July 2022.

https://breakingthenews.net/Article/Zelensky-to-replace-security-service-head/65423949

US Mainstream Media Had Prior Knowledge Of Trump Venezuela Assault, Withheld Coverage

 Via The Cradle

The two largest US newspapers learned in advance of the secret US raid to abduct Venezuelan President Nicolas Maduro, but chose not to publish what they knew to avoid endangering US troops, Semafor reported on 4 January, citing two people familiar with the matter.

Despite their hostility toward US President Donald Trump regarding domestic issues, the New York Times (NYT) and Washington Post cooperated with his administration ahead of the operation to attack Venezuela.

via The Associated Press

US forces deployed more than 150 aircraft to eliminate air defenses, clearing the way for helicopters to insert troops who then moved on to President Maduro’s location.

After Maduro and his wife were abducted, President Trump and top administration officials praised the operation, citing both the lack of US casualties and the total secrecy surrounding it, including from the media.

"The coordination, the stealth, the precision, the very long arm of American justice - all on display in the middle of the night," Pentagon chief Pete Hegseth said.

Trump approved the assault at 10:46 pm Friday. Though aware of the decision, the NYT and Washington Post waited several hours before reporting it because the White House had warned that doing so would expose US troops performing the operation to danger.

However, the decision also showed disregard for the lives of Venezuelans.

US airstrikes accompanying the commando operation killed 40 people, including civilians and military personnel, a senior Venezuelan official told the NYT on Saturday.

One strike targeted a three-story civilian apartment complex in Catia La Mar, a poor coastal area just west of the Caracas airport, killing an 80-year-old woman, Rosa González, and seriously wounding a second person.

Following the airstrikes, US President Donald Trump announced that US forces had "captured" Maduro and his wife, also telling reporters that Maduro had “offered everything” to the US, from Venezuelan oil and natural resources to mediation, according to reporters.

Spokespersons for the White House, the Pentagon, and the Washington Post declined to comment on the conversations between journalists and officials Friday night. A NYT spokesperson did not immediately respond to an inquiry.

https://www.zerohedge.com/political/us-mainstream-media-had-prior-knowledge-trumps-venezuela-assault-withheld-coverage