Aug 31 FDA

Regulatory

AbbVie Receives USFDA Approval for Imbruvica Plus Rituximab as First Chemotherapy-Free Combination Treatment for Waldenström’s Macroglobulinemia Adults

AbbVie announced that the USFDA approved Imbruvica (ibrutinib) plus rituximab (Rituxan) for the treatment of adult patients with Waldenström’s macroglobulinemia (WM), a rare and incurable type of non-Hodgkin’s lymphoma (NHL). With this approval, the Imbruvica prescribing information now includes combination use with rituximab, representing the first and only chemotherapy-free combination treatment specifically indicated for the disease. Imbruvica was first approved as a single agent therapy for WM in January 2015. Imbruvica is a first-in-class Bruton’s tyrosine kinase (BTK) inhibitor jointly developed and commercialized by Pharmacyclics, an AbbVie company, and Janssen.

This new FDA approval is supported by data from the Phase III iNNOVATE (PCYC-1127) trial evaluating Imbruvica in combination with rituximab, versus rituximab alone, in 150 patients with previously untreated and relapsed/refractory WM. At a median follow up of 26.5 months, the study demonstrated a significant improvement in PFS with Imbruvica plus rituximab compared to rituximab alone (30-month PFS rates were 82 percent versus 28 percent, respectively). Patients taking Imbruvica plus rituximab also experienced an 80 percent reduction in relative risk of disease progression or death than those only treated with rituximab.

European Commission Approves Kymriah (tisagenlecleucel), Novartis’ CAR-T Cell Therapy

Novartis has announced that the European Commission (EC) approved Kymriah (tisagenlecleucel, CTL019). The approved indications are for the treatment of pediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse; and for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy.

Kymriah developed in collaboration with the University of Pennsylvania (Penn) is a ground-breaking one-time treatment that uses a patient’s own T cells to fight cancer, and the only chimeric antigen receptor T cell (CAR-T) therapy to receive regulatory approval in the EU for these two distinct B-cell malignancies. Kymriah was also the first CAR-T cell therapy ever approved by the US FDA.

Shire Gets USFDA Approval for Takhzyro (lanadelumab-flyo), a First-of-its-Kind mAb Preventive Treatment for Hereditary Angioedema

Shire has announced that following priority review, the USFDA approved Takhzyro (lanadelumab-flyo) injection, for prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients 12 years of age and older. HAE is a rare, genetic and potentially life-threatening disorder that can result in recurrent attacks of edema (swelling) in various parts of the body. Takhzyro is the only monoclonal antibody (mAb) that provides targeted inhibition of plasma kallikrein, an enzyme which is chronically uncontrolled in people with HAE, to help prevent attacks. The recommended starting dose of Takhzyro is 300 mg every two weeks. A dosing interval of 300 mg every four weeks is also effective and may be considered if the patient is well-controlled (e.g., attack free) for more than six months.

In the Phase III HELP (Hereditary Angioedema Long-term Prophylaxis) Study supporting FDA approval, Takhzyro reduced the number of monthly HAE attacks to an average of 87% (n=27) vs. placebo (n=41) when administered at 300 mg every two weeks and 73% (n=29) vs placebo (n=41) when administered at 300 mg every four weeks. Shire added Takhzyro to its HAE portfolio with the acquisition of Dyax, which was completed in January 2016 in an all cash transaction valued at approximately $5.9 billion.

Roche Receives CNDA Approval for Lung Cancer Drug Alecensa

Roche expanded its presence in China with the approval of its lung cancer drug, Alecensa. It was approved by the China National Drug Administration (CNDA) under a priority review. In China, Alecensa (alectinib) was approved as a monotherapy for patients with anaplastic lymphoma kinase (ALK)-positive, advanced non-small cell lung cancer (NSCLC). The Chinese approval came nine months after the USFDA approved Alecensa in the United States, and eight months after the European Medicines Agency (EMA) approved it in Europe.

NICE Rejects Europe’s First Allogeneic Stem Cell Therapy Alofisel

Cost regulators for the NHS in England and Wales have not approved funding for TiGenix and Takeda’s Alofisel, the first allogeneic stem cell therapy to be approved for use across the European Union for use in Crohn’s patients. The therapy (Cx601) was approved in March to treat complex perianal fistulas in adult patients with nonactive/mildly active luminal Crohn’s disease, when fistulas have shown an inadequate response to at least one conventional or biologic therapy. In clinical trials underpinning the drug’s approval, patients receiving the treatment showing a 44 percent greater probability of achieving combined remission compared to placebo, while a follow-up analysis (at 52 weeks and 104 weeks post-treatment) confirmed sustained efficacy and safety.

Bristol-Myers Squibb’s Opdivo Gets New Approval for Small Cell Lung Cancer

The USFDA gave Bristol-Myers Squibb’s Opdivo (nivolumab) approval for metastatic small cell lung cancer (SCLC) for patients whose cancer has progressed after platinum-based chemotherapy and at least one other type of therapy. The approval was based on data from the SCLC cohort of the ongoing Phase I/II CheckMate -032 clinical trial looking at Opdivo in patients whose disease progressed after platinum-based chemotherapy, the gold standard in chemotherapy. Of the 109 patients who received Opdivo after platinum-based chemo and at least one other previous line of therapy, 12 percent responded to treatment based on a Blinded Independent Central Review (BICR), regardless of expression of PD-L1. Twelve patients (11%) had a partial response and one patient (0.9%) had a complete response. The median duration of response (DOR) was 17.9 months. The drug was discontinued in 10 percent of patients and one dose was withheld in 25 percent of patients because of side effects. Serious side effects were seen in 45 percent of patients.

Eisai and Merck Get USFDA Approval for Lenvima Capsules as First-Line Treatment of Unresectable Hepatocellular Carcinoma

Eisai and Merck announced that the USFDA approved the kinase inhibitor Lenvima (lenvatinib) for the first-line treatment of patients with unresectable hepatocellular carcinoma (HCC). This approval was based on results from REFLECT (Study 304), where Lenvima demonstrated a proven treatment effect on overall survival (OS) by statistical confirmation of non-inferiority, as well as statistically significant superiority and clinically meaningful improvements in progression-free survival (PFS) and objective response rate (ORR) when compared with sorafenib in patients with previously untreated unresectable HCC.

FDA Launches New Draft Guidance for Osteoarthritis Treatments

The USFDA has posted new draft guidance that will guide drug and medical device manufacturers that aim to develop treatments for the underlying pathophysiology and structural progression of osteoarthritis (OA). In its short draft guidance, which was first reported by Regulatory Affairs Professional Society (RAPS), the FDA said that to date, approvals for treatments of osteoarthritis have been based on patient-reported outcome measures that assess pain and function. However, the FDA said that treatments that aimed at the underlying pathophysiology and structural progression of the disease have so far been elusive and constitute an unmet medical need. The guidance will not address the improvement of OA symptoms such as pain or functional impairment. The FDA said those are important outcomes and will be addressed in a future guidance. The new draft guidance was issued days after Regeneron and Teva posted positive Phase III results for fasinumab as a treatment for osteoarthritis (OA) of the knee or hip. As the FDA pushes forward on new OA guidance, it asked for input from drug and device manufacturers to address the considerations.

FDA Rejects Allergan’s Uterine Fibroid Treatment Following EMA Concerns over Liver Damage

The USFDA rejected Allergan’s New Drug Application for ulipristal acetate. Allergan announced that the treatment for abnormal bleeding in women with uterine fibroids was issued a Complete Response Letter by the regulatory agency. Allergan said the FDA cited safety concerns regarding post-marketing reports outside of the United States. While the company did not offer much in the way of information, those post-marketing reports are likely related to the European Medicines Agency (EMA) investigation into questions over whether or not treatment with Esmya, the name UA is marketed under in Europe, led to liver damage in some patients. In Europe, Esmya is approved for the pre-operative and intermittent treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age. The EMA launched its investigation in March and concluded it in May. The committee investigating ruled that the medication may have contributed to some cases of serious liver injury and recommended limited treatment with the medicine. At the end of May, the EMA issued new measures to minimize the risk of liver injury. The measures the EMA instituted include contraindication in women with known liver problems, liver tests before, during and after treatment with Esmya and a notice for patients about the risks of liver damage.

 

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