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The FDA is expected to decide on Tirabrutinib for Relapsed/refractory primary CNS lymphoma by December 18, 2026, under an NDA from Deciphera Pharmaceuticals.
What the FDA is deciding on Tirabrutinib
The FDA is reviewing Deciphera Pharmaceuticals' application to approve Tirabrutinib for Relapsed/refractory primary CNS lymphoma. BTK inhibitor already approved in Japan for this rare brain lymphoma.
Who the Tirabrutinib decision matters to
Primary central nervous system lymphoma (PCNSL) is a fast-growing lymphoma that starts in the brain, spinal cord, eyes or the membranes around them and usually stays there. Treatment relies on high-dose methotrexate, rituximab and sometimes a stem cell transplant, but no drug is specifically approved for it in the United States.
Tirabrutinib, a BTK inhibitor already approved in Japan, has an FDA decision due December 18, 2026 for disease that has come back.
Prevalence: About 5 new cases per million people a year in the US (Ono Pharmaceutical and Deciphera); more than half of patients are 60 or older. See our full Primary Central Nervous System Lymphoma page for current treatments, recruiting trials, and community resources.
How Tirabrutinib works
Tirabrutinib: highly selective, irreversible second-generation Bruton tyrosine kinase (BTK) inhibitor that shuts off B-cell receptor signaling in lymphoma cells. Our full Tirabrutinib profile covers how it works, side effects in plain language, and its trial history.
What an approval or a rejection of Tirabrutinib would mean
An approval starts a second race rather than ending the first one: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. Our guide to the 90 days after a rare disease approval explains the timeline and the moves families can make on day 1.
A complete response letter would mean the FDA declined to approve in the application's current form. The letter describes what the FDA wants addressed, and the company then decides whether to resubmit, run more studies or stop. Either way, this page updates with the outcome and what it means.
Where Primary Central Nervous System Lymphoma treatment stands today
Read our full Primary Central Nervous System Lymphoma treatment summary
The standard first treatment is high-dose methotrexate given in the hospital, usually combined with rituximab and other chemotherapy drugs such as cytarabine and temozolomide, followed in fitter patients by high-dose chemotherapy with an autologous stem cell transplant, which has been shown to lengthen survival. Whole-brain radiation can control the disease but can also damage thinking, memory and speech.
For people who cannot tolerate high-dose methotrexate, which includes most patients over 75 and people with significantly reduced kidney function, lenalidomide with rituximab is the most frequently used alternative. The National Cancer Institute also lists ibrutinib, nivolumab and CAR T cell therapy as options, but none of these, and no other drug, has been specifically approved by the FDA for PCNSL.
The first candidate for that approval is tirabrutinib, an oral BTK inhibitor discovered by Ono Pharmaceutical and brought to the US by its subsidiary Deciphera.
The FDA accepted the application under the accelerated approval pathway with a decision due December 18, 2026, based on the Phase 2 PROSPECT study (NCT04947319), in which 48 patients with relapsed or refractory PCNSL took the drug once a day: 67% responded, 44% had a complete response, and the median time to response was 1 month, with anemia, rash, fatigue and low blood counts the most common side effects.
Tirabrutinib has been approved for this use in Japan since March 2020, where it is sold as Velexbru. A confirmatory Phase 3 trial (NCT07104032) is recruiting.
A neuro-oncologist or lymphoma specialist is the right person to ask about trials; the Lymphoma Research Foundation's helpline (800-500-9976) and the nurses at Blood Cancer United's Clinical Trial Support Center can help you find one and read the eligibility rules.
The Primary Central Nervous System Lymphoma page has every treatment and trial →Meanwhile, 71 Primary Central Nervous System Lymphoma studies are open or about to open
Whatever the FDA decides here, research on Primary Central Nervous System Lymphoma does not stop. The most recently updated:
We'll email you when the decision is public, with what it means in plain language, and when new trials open for Primary Central Nervous System Lymphoma. Unsubscribe anytime.
- Trial Alerts, when a new trial opens or a trial changes
- Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
Frequently asked questions
When will the FDA decide on Tirabrutinib?
The FDA's target date (PDUFA date) for Tirabrutinib in Relapsed/refractory primary CNS lymphoma is December 18, 2026.
Who disclosed it: Deciphera Pharmaceuticals. The FDA does not publish these dates.
Can it move: the FDA can act early, and a major amendment to the application can push the date back 3 months.
Source: Deciphera announcement
What is Tirabrutinib being reviewed for?
Deciphera Pharmaceuticals submitted an NDA for Tirabrutinib in Relapsed/refractory primary CNS lymphoma.
What to know: BTK inhibitor already approved in Japan for this rare brain lymphoma.
How it works: Highly selective, irreversible second-generation Bruton tyrosine kinase (BTK) inhibitor that shuts off B-cell receptor signaling in lymphoma cells.
More: the Tirabrutinib drug page explains it in plain language.
Is there already an FDA-approved treatment for Primary Central Nervous System Lymphoma?
No. No medicine's current FDA label names Primary Central Nervous System Lymphoma, checked October 21, 2026.
Check: the Primary Central Nervous System Lymphoma page also searches FDA drug labels for approved treatments.
What did the Tirabrutinib trials show?
PROSPECT (NCT04947319) is a Phase 2 study with 2 parts. Part A enrolled 48 US patients with relapsed or refractory primary CNS lymphoma who took tirabrutinib alone.
More: the Tirabrutinib drug page has the full trial history and sources.
What are the side effects of Tirabrutinib?
In PROSPECT, 56.3% of the 48 patients had a grade 3 or higher treatment-emergent adverse event, and 75.0% had an adverse event of any grade that was considered related to the drug. The most frequent drug-related events were anemia (18.8%), a measles-like rash (16.7%), fatigue (14.6%), low neutrophil counts (14.6%), low lymphocyte counts (14.6%), itching (14.6%) and other rash (14.6%).
More: the Tirabrutinib drug page has the safety details reported so far.
Can I get Tirabrutinib before the FDA decides?
Usually only through a clinical trial or an expanded access program, and the company decides whether to offer one.
Trials: 71 Primary Central Nervous System Lymphoma studies are open or about to open. See them.
Check your fit: Match Me compares your details with every open trial.
Expanded access: your doctor can ask Deciphera Pharmaceuticals directly. Registered programs are listed on ClinicalTrials.gov.
How much will Tirabrutinib cost?
Deciphera Pharmaceuticals has not announced a price. Drugmakers usually set it around approval.
Help paying: drugmaker programs and charity funds usually open at launch.
Find help: the patient assistance finder lists programs by insurance type.
What happens after the Tirabrutinib decision?
This page updates with the outcome the day it is announced.
If approved: getting the drug still takes time. Specialty pharmacy setup, insurance review and patient assistance often take weeks. Our guide covers what families can do on day 1.
If not approved: a complete response letter means the FDA declined the application in its current form. Many are about manufacturing or how data were presented, and companies often resubmit.
Where this date comes from
The FDA does not publish PDUFA dates; companies disclose them. This one comes from Deciphera announcement. Dates can move, and the FDA can act early or late. This page rechecks against our calendar, which is re-verified daily.