Pfizer and Spark Therapeutics Lead Drug Approval News

A roundup of the latest drug approvals, including from the pharmaceutical majors, featuring news from Pfizer and Spark Therapeutics.

Editor’s Note: This article is updated on a continuous basis for news announced from Wednesday, December 13, 2017 to Tuesday, January 9, 2017.

FDA OKs New Use for Pfizer’s Leukemia Drug Bosulif
The US Food and Drug Administration (FDA) has approved Pfizer’s supplemental new drug application (sNDA) to expand the indication for Bosulif (bosutinib) to include adult patients with newly-diagnosed chronic phase Philadelphia chromosome-positive chronic myelogenous leukemia (Ph+ CML).

Bosulif is an oral, once-daily, tyrosine kinase inhibitor, which inhibits the Bcr-Abl kinase that promotes CML; it is also an inhibitor of Src-family kinases.

The sNDA was reviewed and approved under the FDA’s priority review and accelerated approval programs based on molecular and cytogenetic response rates. Continued approval for this indication may be contingent upon verification and confirmation of clinical benefit in an ongoing long-term follow up trial. Bosulif was first approved in September 2012 in the US for treating adult patients with chronic, accelerated, or blast phase Ph+ CML with resistance or intolerance to prior therapy.

Pfizer and Avillion, a London, UK-based company focused on increasing research and development output, entered into an exclusive collaborative development agreement in 2014 to conduct the trial for Bosulif. Under the agreement, Avillion provided funding and conducted the trial to generate the clinical data used to support this application and other potential regulatory filings for marketing authorization for Bosulif as first-line treatment for patients with chronic phase Ph+ CML. With this approval, Avillion is eligible to receive milestone payments from Pfizer. Pfizer retains all rights to commercialize the drug globally.

Source: Pfizer

FDA OKs New Use for Pfizer’s Arthritis Drug Xeljanz
The US Food and Drug Administration (FDA) has approved a new use for Pfizer’s Xeljanz (tofacitinib) 5-mg twice-daily and Xeljanz XR extended-release 11-mg once daily for treating adult patients with active psoriatic arthritis (PsA) who have had an inadequate response or intolerance to methotrexate or other disease-modifying antirheumatic drugs (DMARDs).

Xeljanz/Xeljanz XR is a janus kinase inhibitor that was previously approved by the FDA for treating moderate-to-severe rheumatoid arthritis (RA). Xeljanz/Xeljanz XR (tofacitinib) is indicated for treating adult patients with moderately to severely active RA who have had an inadequate response or intolerance to methotrexate. It may be used as monotherapy or in combination with methotrexate or other nonbiologic DMARDs.

Source: Pfizer

FDA OKs Spark Therapeutics’ Gene Therapy
The US Food and Drug Administration has approved Luxturna (voretigene neparvovec-rzyl), a gene therapy, from Spark Therapeutics, a Philadelphia, Pennsylvania-based gene-therapy company, for treating patients with an inherited form of vision loss that may result in blindness.

Luxturna is a directly administered gene therapy designed to target a disease caused by mutations in a specific gene. The drug is approved for treating patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss and may cause complete blindness in certain patients.

The FDA granted this application priority review and breakthrough therapy designations. Luxturna also received orphan-drug designation, which provides incentives to assist and encourage developing drugs for rare diseases.

Spark Therapeutics is receiving a rare pediatric disease priority review voucher under a program intended to encourage development of new drugs and biologics for preventing and treating rare pediatric diseases. A voucher can be redeemed by a sponsor at a later date to receive priority review of a subsequent marketing application for a different product.

To further evaluate the long-term safety, the company plans to conduct a post-marketing observational study involving patients treated with Luxturna.

Spark Therapeutics is partnered with Pfizer for gene-therapy product development. In 2014, the companied formed an agreement to develop SPK-FIX, a program incorporating a bio-engineered adeno-associated virus (AAV) vector for the potential treatment of hemophilia B. In 2016, the companies were granted breakthrough therapy designation by the FDA for SPK-9001, the companies’ lead investigational candidate for hemophilia B.

Source: FDA


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