Recent FDA Approvals

June 13, 2018: Pembrolizumab

FDA granted accelerated approval to pembrolizumab (Keytruda, Merck) for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after two or more prior lines of therapy.

It is an IgG4 isotype antibody that blocks programmed cell death 1 (PD-1) receptor of lymphocytes.

Approval was based on data from 53 patients with relapsed or refractory PMBCL enrolled in a multicenter, open-label, single-arm trial, KEYNOTE‑170 (NCT02576990).

Pembrolizumab is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Read @ FDA website


June 8, 2018: Venetoclax

FDA granted regular approval to venetoclax (VENCLEXTA, AbbVie Inc. and Genentech Inc.) for patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), with or without 17p deletion, who have received at least one prior therapy.

Approval was based on MURANO (NCT02005471), a randomized (1:1), multicenter, open-label trial of venetoclax with rituximab (VEN+R) versus bendamustine with rituximab (B+R) in 389 patients with CLL who had received at least one prior line of therapy.

In 2015, the United States Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to venetoclax for subjects with CLL who have relapsed or have been refractory to previous treatment and have the 17p deletion genetic mutation.

On April 11, 2016, the FDA approved venetoclax for use in those with CLL who have 17p deletion (deletion located on the chromosome 17 short arm) and who have been treated with at least one prior therapy.

Venetoclax blocks the anti-apoptotic B-cell lymphoma-2 (Bcl-2) protein, leading to programmed cell death of CLL cells.

Read @ FDA website


May 21, 2018: Avatrombopag

FDA approved avatrombopag (Doptelet, AkaRx Inc.) for thrombocytopenia in adults with chronic liver disease scheduled to undergo a procedure.

Approval was based on two international, identically designed, randomized, double-blind, placebo-controlled trials, ADAPT-1 and ADAPT-2.


May 1, 2018: Tisagenlecleucel

Kymriah (tisagenlecleucel) is a CD19-directed genetically modified autologous T cell immunotherapy.

FDA approved tisagenlecleucel (KYMRIAH, Novartis Pharmaceuticals Corp.) a CD19-directed genetically modified autologous T-cell immunotherapy, for adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma.

Approval was based on a single-arm, open-label, multi-center, phase 2 trial (JULIET, NCT02445248) in adults with relapsed or refractory DLBCL and DLBCL after transformation from follicular lymphoma. Patients received a single infusion of tisagenlecleucel following completion of lymphodepleting chemotherapy.

The recommended dose of tisagenlecleucel for relapsed or refractory adult DLBCL is 0.6 to 6.0 x 108 CAR-positive viable T cells. Tisagenlecleucel is not indicated for the treatment of patients with primary central nervous system lymphoma.

NOTE : Was approved in Aug 2017 for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.

Read @ FDA website


April 17, 2018: Fostamatinib

FDA approved fostamatinib disodium hexahydrate tablets (TAVALISSE, Rigel Pharmaceuticals, Inc.) for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

The drug is administered orally as a disodium hexahydrate salt, and is a prodrug of the active compound tamatinib (R-406), which is an inhibitor of the enzyme spleen tyrosine kinase (Syk), hence it is an syk inhibitor.

Approval was based on two identical, double-blind, placebo-controlled trials, FIT-1 (NCT02076399) and FIT-2 (NCT02076412) that enrolled a total of 150 patients with persistent or chronic ITP who had an insufficient response to previous treatment, which included corticosteroids, immunoglobulins, splenectomy, and/or a thrombopoietin receptor agonist.

Read @ FDA website


 

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