BMS, GSK, Janssen, and Sanofi Lead Pipeline News
A roundup of the latest market developments from the pipelines of the pharmaceutical majors and other related news, featuring news from Bristol-Myers Squibb, GlaxoSmithKline, Janssen, Sanofi, Biogen, Gilead, AstraZeneca, and Novartis.
Editor’s Note: This article is updated on a continuous basis for news announced from Wednesday September 21, 2016 to Tuesday September 27, 2016.
EMA Validates BMS Application to Extend Opdivo Indications
The European Medicines Agency (EMA) has validated Bristol-Myers Squibb’s Type II variation application, which seeks to extend the current indications for its immuno-oncology drug, Opdivo (nivolumab), to include treatment of locally advanced unresectable or metastatic urothelial carcinoma in adults after failure of prior platinum-containing therapy. Validation of the application confirms the submission is complete and begins the EMA’s centralized review process.
Opdivo currently has regulatory approval in 54 countries including the US, Japan, and countries in the European Union. Opdivo is projected to be a blockbuster drug by some analysts. In 2015, it had global sales of $942 million, and a recent Thomson Reuters analysis projects that 2019 sales will reach nearly $8.9 billion.
Opdivo is a human antibody designed to alleviate immune suppression. In the US, it is indicated for treating patients with non-small cell lung cancer with progression on or after platinum-based chemotherapy; as a single agent for treating BRAF V600 wild-type unresectable or metastatic melanoma and as a single agent for treating BRAF V600 mutation-positive unresectable or metastatic melanoma. It is also approved in combination with Yervoy (ipilimumab) for treating unresectable or metastatic melanoma and for treating advanced renal cell carcinoma that has received prior anti-angiogenic therapy. It is also indicated for treating patients with classical Hodgkin lymphoma that has relapsed or progressed after autologous hematopoietic stem cell transplantation and post-transplantation brentuximab vedotin.
Source: Bristol Myers Squibb
GSK, Janssen Seek FDA Approval for Rheumatoid Arthritis Drug
Janssen Biotech, a Johnson & Johnson company, and GlaxoSmithKline have submitted a biologics license application (BLA) to the US Food and Drug Administration (FDA) seeking approval of a subcutaneous formulation of sirukumab, a human anti-interleukin (IL)-6 monoclonal antibody, for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA) who have failed or are intolerant to one or more disease-modifying antirheumatic drugs.
Sirukumab is being co-developed for RA as part of a collaboration between GSK and Janssen Biologics. Sirukumab is an investigational human monoclonal immunoglobulin G1 kappa antibody that selectively binds to the IL-6 cytokine. Approval is being sought for the subcutaneous formulation of sirukumab in two presentations, a single-dose prefilled syringe and a single-dose autoinjector.
A regulatory submission to the European Medicines Agency (EMA) was announced on September 12, 2016. Sirukumab is currently not approved as a treatment for any indication anywhere in the world.
In December 2011, GSK and Janssen entered into a licensing and co-development agreement with respect to sirukumab. Under the terms, GSK has exclusive rights to commercialize sirukumab in North, Central and South America, while Janssen retains commercialization rights in the rest of the world, including such territories as Europe, Middle East and Africa, and Asia Pacific. Global profit will be shared equally between the two companies. Prior to the agreement, Janssen had been developing sirukumab for RA.
Janssen is responsible for the FDA and EMA regulatory files. The collaboration gives both companies the option to investigate sirukumab for other indications beyond RA.
Janssen Gets FDA OK for Crohn’s Disease Drug, Seeks Expanded Indication for Lymphoma Drug
Janssen Biotech, a Johnson & Johnson company, has received approval from the US Food and Drug Administration (FDA) for Stelara (ustekinumab) for moderately to severely active Crohn’s disease in adults (18 years or older) who have failed or were intolerant to treatment with immunomodulators or corticosteroids but never failed treatment with a tumor necrosis factor (TNF) blocker, or who failed or were intolerant to treatment with one or more TNF blockers.
Janssen said it will work closely with payers, providers and pharmacy benefit managers to ensure Stelara is broadly accessible and affordable for patients, and that the cost for payers is competitive with currently available biologic therapies for Crohn’s disease.
In related news, Janssen Biotech and AbbVie have submitted a supplemental new drug application to the US FDA for Imbruvica (ibrutinib) for treating patients with marginal zone lymphoma (MZL). Imbruvica is jointly developed and commercialized by Pharmacyclics, an AbbVie company, and Janssen Biotech. AbbVie acquired Pharmacyclics in 2015.
Imbruvica is currently approved to treat patients with chronic lymphocytic leukemia/small lymphocytic lymphoma, including patients with 17p deletion, patients with mantle cell lymphoma who have received at least one prior therapy, and patients with Waldenström’s macroglobulinemia.
Sanofi, Regeneron Get Priority Review for Skin Disease Drug
Sanofi and Regeneron Pharmaceuticals received priority review from the US Food and Drug Administration (FDA) for the biologics license application for dupilumab for adult patients with inadequately controlled moderate-to-severe atopic dermatitis, a chronic inflammatory skin disease. The target action date for the application is March 29, 2017.
Dupilumab is currently under clinical development, and its safety and efficacy have not been fully evaluated by any regulatory authority. If approved, dupilumab would be commercialized by Regeneron and Sanofi Genzyme, the specialty care global business unit of Sanofi.
GSK Submits Subcutaneous Formulation for Lupus Disease
GlaxoSmithKline has filed regulatory submissions in the US and Europe for Benlysta (belimumab) for approval as a subcutaneous formulation in patients with active, autoantibody-positive systemic lupus erythematosus (SLE). The submissions comprise of a biologics license application to the US Food and Drug Administration and an extension marketing authorization application to the European Medicines Agency.
The BLA and extension MAA are seeking approval for the Benlysta subcutaneous formulation in two presentations, a single-dose prefilled syringe and a single-dose autoinjector. Regulatory filings in other countries are planned during the course of 2016 and 2017. The subcutaneous formulation of Benlysta is currently not approved for use.
Biogen Submits NDA for Spinal Muscular Atrophy Drug
Biogen has completed its rolling submission of a new drug application (NDA) to the US Food and Drug Administration for nusinersen, an investigational treatment for spinal muscular atrophy (SMA). Biogen has also applied for priority review which, if granted, would shorten the review period of nusinersen following the agency’s acceptance of the NDA.
In addition to the NDA filing with the FDA, Biogen plans to submit a marketing authorization application for nusinersen to the European Medicines Agency (EMA) in the coming weeks. The EMA’s Committee for Medicinal Products for Human Use (CHMP) recently granted accelerated assessment to nusinersen. Biogen will initiate regulatory filings in other countries in the coming months.
Nusinersen is an investigational antisense oligonucleotide being developed for SMA treatment. Both the US and European Union (EU) regulatory agencies have granted special status to nusinersen, including fast-track designation in the US and orphan-drug designation in both the US and EU.
Lundeck’s Alzheimer’s Disease Drug Fails Phase III Trial
H. Lundbeck A/S has announced that its investigational drug, idalopirdine, for the symptomatic treatment of patients with mild-to-moderate Alzheimer’s disease, failed to meet its primary endpoint in a Phase III clinical study. Further analysis of the data from the study is ongoing. The drug remains in two other Phase III studies that are currently ongoing and will be continued, with data expected in the first quarter of 2017.
Source: H. Lundeck A/S
AstraZeneca Withdraws MAA in Europe for Cancer Drug
AstraZeneca has decided to withdraw its marketing authorization application (MAA) submitted to the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) in June 2015 for cediranib in combination with platinum-based chemotherapy followed by maintenance monotherapy for the treatment of adult patients with platinum-sensitive relapsed ovarian cancer. The decision to withdraw the MAA was based on outstanding health authority questions that remain at this late stage of the review process, AstraZeneca said. The company has not made additional regulatory submissions for cediranib in this indication in any other markets.
Cediranib remains a part of AstraZeneca’s ovarian cancer medicine pipeline, and this decision does not affect the ongoing primary development program testing cediranib as a combination treatment alongside the company’s existing and potential medicines.
Gilead Terminates Antibody Clinical Study
Gilead Sciences has announced that it is stopping its combined Phase II/III clinical study of GS-5745, an investigational anti-MMP9 antibody, among patients with moderately to severely active ulcerative colitis. This decision follows a planned interim analysis of unblinded efficacy and safety data that led to the recommendation that the study be terminated early due to meeting the pre-specified futility and efficacy criteria. No safety concerns were noted in this interim analysis.
Gilead has also reviewed the data and determined that there is insufficient evidence of a treatment benefit from using GS-5745. Separately, a Phase III study is ongoing in patients with gastric cancer as well as a Phase II study in patients with gastric cancer in combination with nivolumab and additional Phase II studies in moderately to severely active Crohn’s disease, rheumatoid arthritis, and cystic fibrosis. These studies will continue as planned.
Source: Gilead Sciences
Sanaria’s Malaria Vaccine Gets FDA Fast Track
Sanaria, a Rockville, Maryland-based biotechnology company, has been granted fast-track designation by the US Food and Drug Administration its preventative vaccine for malaria, Sanaria PfSPZ Vaccine. The vaccine is in clinical trials in subjects ranging from six-month old infants to adults in the US, Europe, and Africa. These trials are intended to finalize an immunization regimen to be taken forward into Phase 3 clinical trials that will, if successful, provide necessary data for licensing the vaccine.
Clinical trials are in progress in Tanzania, Kenya, Mali, Burkina Faso, Germany, and two sites in the US and are intended to begin soon in Equatorial Guinea.
FDA Approves Janssen’s Diabetes Combo Therapy
Janssen Pharmaceuticals, a Johnson & Johnson company, has received approval from the US Food and Drug Administration for Invokamet XR, a once-daily, fixed-dose combination therapy of canagliflozin and metformin hydrochloride extended-release, for first-line use as an adjunct to diet and exercise to improve blood glucose control in adults with Type 2 diabetes when treatment with the two medications is appropriate.
The approved indication for Invokamet XR aligns with current Type 2 diabetes treatment guidelines from the American Association of Clinical Endocrinologists and American College of Endocrinology and from the American Diabetes Association, which recommend dual therapy for patients with higher A1C
levels. Invokamet XR is available in four dosages in tablets containing canagliflozin 50 milligrams (mg) or 150 mg and metformin XR 500 mg or 1000 mg.
Source: Johnson & Johnson
Novartis Gets FDA OK on Expanded Use of Ilaris
Novartis has been granted three simultaneous approvals by the US Food and Drug Administration (FDA) for the expanded use of Ilaris (canakinumab) to treat three rare and distinct types of periodic fever syndromes. Ilaris is an FDA-approved biologic treatment for tumor necrosis factor-receptor associated periodic syndrome, hyperimmunoglobulin D syndrome/mevalonate kinase deficiency, and familial Mediterranean fever. All three conditions are part of a group of rare autoinflammatory diseases called periodic fever syndromes, or hereditary periodic fevers.
Ilaris is already approved and marketed in the US as a treatment for another periodic fever syndrome condition, cryopyrin-associated periodic syndromes (CAPS), and another autoinflammatory condition, systemic juvenile idiopathic arthritis. In August 2016, the European Commission approved Ilaris for a license extension to also treat patients with adult-onset Still’s disease.
Ilaris is a selective, human monoclonal antibody that inhibits interleukin-1 (IL-1) beta. It is also approved in more than 70 countries, including in the European Union, Switzerland, Canada, and Japan for the treatment of the periodic fever syndrome CAPS. In the US, Ilaris is approved for two subtypes of CAPS: Muckle-Wells syndrome (MWS) and familial cold autoinflammatory syndrome. The approved indications may vary depending upon the individual country.