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Regulatory Focus™ > News Articles > 2020 > 6 > FDA Approvals Roundup: Tazverik, Crysvita, Xpovio

FDA Approvals Roundup: Tazverik, Crysvita, Xpovio

Posted 24 June 2020 | By Renee Matthews 

FDA Approvals Roundup: Tazverik, Crysvita, Xpovio

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
 
New indications
Tazverik extended as therapy for mutated follicular lymphoma
Epizyme’s Tazverik (tazemetostat) has been granted accelerated approval for treating adult patients with relapsed or refractory follicular lymphoma whose tumors are positive for an EZH2 mutation, as detected by an FDA-approved companion diagnostic, the cobas EZH2 Mutation Test (Roche Molecular Systems).
 
The EZH2 inhibitor is approved for previously treated patients and those with no alternative treatment options in this expanded indication. Earlier this year, it received accelerated approval for treatment of adults and pediatric patients aged 16 years or older with metastatic or locally advanced epithelioid sarcoma who are not eligible for complete resection.
 
Approval for the indication for follicular lymphoma was based on two open-label, single-arm cohorts of a multicenter trial in patients whose follicular lymphoma persisted after at least two previous systemic therapies. EZH2 mutations were identified prospectively, using the cobas test. Patients received Tazverik twice daily until confirmed disease progression or unacceptable toxicity.
 
Efficacy was based on overall response rate (ORR) and duration of response (DOR). In 42 patients with EZH2-mutant follicular lymphoma, ORR was 69% (12% complete responses, 57% partial) and median DOR was 10.9 months. In 53 patients with EZH2 wild-type disease, ORR was 34% (4% complete, 30% partial) and median DOR was 13 months.
 
The indications both received accelerated approval based on ORR and DOR, so continued approval for the indications may be contingent on verification and description of clinical benefit in a confirmatory trial or trials.
 
The current application was granted priority review and fast track designation.
 
Crysvita approved as first therapy for rare tumor-related bone disease
Ultragenyx Pharmaceutical’s Crysvita (burosumab-twza) injection has been approved to treat patients aged 2 years or older who have tumor-induced osteomalacia (TIO), a rare disease in which tumors release a substance that lowers blood phosphate levels, causing bone weakening and softening.
 
The monoclonal antibody acts by boosting blood phosphate levels to prevent bone deterioration. It was originally approved in 2018 for treating adults and children aged 6 months and older with X-linked hypophosphatemia, a rare inherited form of rickets that lowers blood phosphate levels and impairs bone development in children and teenagers.
 
The original biologics license application for the drug was granted breakthrough therapy and fast track designations. The therapy also received orphan drug status.
 
XPOVIO gets new indication for treating aggressive DLBCL
Karyopharm Therapeutics’ XPOVIO (selinexor) has been granted accelerated approval for the treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, in adult patients who have received at least two lines of systemic therapy.
 
This indication was approved under accelerated approval based on response rate, so continued approval for these indications may be contingent on verification and description of clinical benefit in confirmatory trials. The application was granted priority review and fast track designation.
 
In July 2019, Karyopharm received accelerated approval for the drug in combination with dexamethasone for previously treated adults with relapsed or refractory multiple myeloma. The current expanded indication makes the first-in-class nuclear export inhibitor the first drug to be approved by the FDA for use in both multiple myeloma and DLBCL. The company has also submitted a marketing authorization application to the European Medicines Agency, requesting conditional approval of the therapy for the same relapsed/refractory multiple myeloma indication.

Tags: FDA, US

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