Amgen, BMS, Novartis, and Roche Lead Pipeline News
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A roundup of the latest market developments from the pipelines of the pharmaceutical majors and other related news, featuring news from Amgen, BMS, Novartis, and Roche.

Editor’s Note: This article is updated on a continuous basis for news announced from Wednesday March 15, 2017 to Tuesday April 11, 2017.

EMA Grants Orphan Drug Status to AstraZeneca’s Autoimmune Drug
The European Medicines Agency (EMA) has granted AstraZeneca and MedImmune, AstraZeneca’s global biologics research and development arm, orphan designation for inebilizumab, a drug candidate for treating neuromyelitis optica spectrum disorder (NMOSD), a rare, life-threatening autoimmune disease of the central nervous system. Developed by MedImmune, inebilizumab is currently in Phase IIb clinical development for NMOSD.

Orphan designation is a status assigned to a medicine intended for use in rare diseases. To be granted orphan designation by the EMA, a medicine must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening and has a prevalence of less than five people in 10,000 in the European Union. Additionally, the intended medicine must aim to provide significant benefit to those affected by the condition. Orphan designation provides companies with development and market exclusivity incentives for designated compounds and medicines. Orphan designation is conferred following a positive opinion by the EMA’s Committee for Orphan Medicinal Products (COMP).

AstraZeneca also received orphan drug designation for inebilizumab in March 2016 from the US Food and Drug Administration. Inebilizumab is a humanized, monoclonal antibody that binds with affinity to CD19, a protein expressed on a broad range of B cells, including certain B cells called plasmablasts, and depletes them, according to AstraZeneca. Inebilizumab is currently being evaluated in a global clinical trial in neuromyelitis optica (NMO)/NMOSDs. In NMOSD, the body’s immune system attacks healthy cells, most commonly in the optic nerves and spinal cord, and may cause severe muscle weakness and paralysis, loss of vision, respiratory failure, problems with bowel and bladder function, and neuropathic pain, according to AstraZeneca.

Source: AstraZeneca


Amgen Files in Europe and US to Expand Cancer Drug Indication
Amgen has submitted a supplemental biologics license application to the US Food and Drug Administration (FDA) and an application for a variation to the marketing authorization to the European Medicines Agency (EMA) for Xgeva(denosumab), seeking to expand the currently approved Xgeva indication for preventing skeletal-related events (SREs) in solid tumors to include multiple myeloma. The applications to the FDA and EMA include new data from a Phase III clinical trial in multiple myeloma.

Xgeva is a fully human monoclonal antibody currently indicated for preventing SREs in patients with bone metastases from solid tumors and for treating giant-cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity. In the US, Xgeva is also indicated for treating hypercalcemia of malignancy refractory to bisphosphonate therapy.

Source: Amgen


FDA Accepts BMS Filing for Extended Opdivo Indication
The US Food and Drug Administration (FDA) has accepted a supplemental biologics license application filed by Bristol-Myers Squibb (BMS) seeking to extend the use of Opdivo (nivolumab), BMS’ programmed death-1 (PD-1) immune checkpoint inhibitor, to patients with mismatch repair deficient (dMMR) or microsatellite instability high (MSI-H) metastatic colorectal cancer (CRC). The FDA granted the application priority review and has set the action date on August 2, 2017. Opdivo, approved by the FDA in 2014, is a blockbuster anti-cancer drug for BMS with 2016 sales of $3.77 billion.

In the US, Opdivo is indicated for treating BRAF V600 mutation-positive unresectable or metastatic melanoma and BRAF V600 wild-type unresectable or metastatic melanoma as a single agent; unresectable or metastatic melanoma in combination with Yervoy (ipilimumab); metastatic non-small cell lung cancer; advanced renal cell carcinoma; classical Hodgkin lymphoma; recurrent or metastatic squamous cell carcinoma of the head and neck; and locally advanced or metastatic urothelial carcinoma.

Source: Bristol-Myers Squibb


EMA Positive on Extended Indication for BMS’ Cancer Drug
Bristol-Myers Squibb (BMS) had received a recommendation for approval from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for use of Opdivo (nivolumab), a programmed death-1 (PD-1) immune checkpoint inhibitor, as monotherapy for treating squamous cell cancer of the head and neck (SCCHN) in adults progressing on or after platinum-based therapy. This CHMP recommendation will be reviewed by the European Commission (EC). Opdivo, a blockbuster drug for BMS with 2016 sales of $3.77 billion, is already approved by the EC for six indications in four distinct tumor types.

Opdivo first received regulatory approval in July 2014 and is currently approved in more than 60 countries, including the US, the European Union (EU), and Japan. In October 2015, the immuno-oncology combination regimen of Opdivo and Yervoy (ipilimumab) received regulatory approval for treating metastatic melanoma and is currently approved in more than 50 countries, including the US and the EU.

US Food and Drug Administration-approved indications for Opdivo in the US include: as a single agent for treating BRAF V600 mutation-positive unresectable or metastatic melanoma; as a single agent for treating BRAF V600 wild-type unresectable or metastatic melanoma; in combination with Yervoy for treating unresectable or metastatic melanoma; treating metastatic non-small cell lung cancer; treating advanced renal cell carcinoma; treating classical Hodgkin lymphoma; treating metastatic SCCHN; and treating locally advanced or metastatic urothelial carcinoma.

Source: Bristol-Myers Squibb 


FDA Accepts J&J’s Filing for New Cancer Drug Indication
The US Food and Drug Administration (FDA) has accepted for review a supplemental new drug application filed by Janssen Research & Development, a Johnson & Johnson company, seeking approval for an additional indication for its anti-cancer drug, Imbruvica (ibrutinib), in treating chronic graft-versus-host-disease (cGVHD) after failure of one or more lines of systemic therapy.

Imbruvica, which is jointly developed and commercialized by Janssen Biotech, also a Johnson & Johnson company, and Pharmacyclics, an AbbVie company, is a kinase inhibitor approved for treating mantle cell lymphoma and chronic lymphocytic leukemia. GVHD is a potential life-threatening consequence of stem cell or bone marrow transplant.

Source: Johnson & Johnson


FDA Extends Action Date for Merck & Co.’s Keytruda for New Cancer Indication
The US Food and Drug Administration (FDA) has extended by three months the Prescription Drug User Fee Act (PDUFA) target action date for Merck & Co.’s supplemental biologics license application for Keytruda (pembrolizumab), the company’s anti-programmed death-1 therapy, for previously treated advanced microsatellite instability-high (MSI-H) cancer.

The extension is based on additional data and analyses recently submitted by Merck to the FDA related to the pending application. The submission of additional data is considered a major amendment to the sBLA under PDUFA, which requires the extension of the target action date, which is now June 9, 2017.

Keytruda is indicated in the US for treating unresectable or metastatic melanoma, metastatic non-small cell lung cancer, recurrent or metastatic head and neck squamous cell carcinoma, and refractory classical Hodgkin lymphoma.

Source: Merck & Co.


Merck & Co. Gains Positive EMA Opinion on Keytruda for Hodgkin Lymphoma
Merck & Co. has received a positive opinion from the Committee for Medicinal Products for Human Use of the European Medicines Agency recommending approval of Keytruda (pembrolizumab), the company’s anti-programmed death-1 therapy, for treating relapsed or refractory classical Hodgkin lymphoma. The recommendation will now be reviewed by the European Commission for marketing authorization in the European Union. A decision on approval is expected in the second quarter of 2017.

In the US, Keytruda is approved for treating unresectable or metastatic melanoma, metastatic non-small cell lung cancer, recurrent or metastatic head and neck squamous cell carcinoma, and refractory classical Hodgkin lymphoma.

Source: Merck & Co.


FDA Accepts Novartis’ Filing for CAR T Leukemia Drug
Novartis’ biologics license application (BLA) for tisagenlecleucel-T, an investigational chimeric antigen receptor T cell (CAR-T) therapy, has been accepted and granted priority review by the US Food and Drug Administration for treating pediatric and young-adult relapsed and refractory (r/r) B-cell acute lymphoblastic leukemia (ALL).

CAR-T therapies are manufactured for each individual patient. During the treatment process, T cells are drawn from a patient’s blood and reprogrammed in the laboratory to create T cells that are genetically coded for that individual patient’s cancer cells and other B-cells expressing a particular antigen.

Novartis plans additional filings for tisagenlecleucel-T in the US and European Union markets later this year, including a BLA with the FDA for treating adults with r/r diffuse large B-cell lymphoma (DLBCL) and applications for marketing authorization with the European Medicines Agency in r/r B-cell ALL and r/r DLBCL.

Source: Novartis


EMA Positive on Updated Labeling for Novo Nordisk’s New Insulin
Novo Nordisk has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP), under the European Medicines Agency recommending an update of the label for Tresiba (insulin degludec), a diabetes medicine, to include data from clinical trials in which Tresiba demonstrated clinically relevant reductions in hypoglycemia compared with insulin glargine in Type 1 and Type 2 diabetes.

The CHMP positive opinion is now referred for final action to the European Commission, which grants approval in the European Union. Novo Nordisk expects to receive an updated marketing authorization in the second quarter of 2017.

Tresiba received regulatory approval in September 2012 and has since been approved in more than 80 countries globally. It was approved in the US by the US Food and Drug Administration in September 2015. The drug is now commercially available in more than 50 countries.

Source: Novo Nordisk


Novo Nordisk Gains Positive EMA Opinion on Hemophilia Drug
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion for Novo Nordisk’s Refixia (nonacog beta pegol, N9-GP), recommending marketing authorization for treating hemophilia B.

The CHMP recommends Refixia to be indicated for prophylaxis and on-demand treatment of bleeding as well as for surgical procedures. Refixia is an extended half-life factor IX molecule for replacement therapy in patients with hemophilia B.

Source: Novo Nordisk 


EMA Positive on Pfizer’s Meningococcal Vaccine
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending that Pfizer’s Trumenba (meningococcal group B vaccine) be granted marketing authorization in the European Union for active immunization of individuals 10 years and older to prevent invasive meningococcal disease caused by Neisseria meningitidis serogroup B (MenB). The CHMP’s opinion will be sent to the European Commission for a final decision.

Trumenba, a sterile suspension composed of two recombinant lipidated factor H binding protein variants from N. meningitidis serogroup B, was introduced in the US in October 2014, where it is indicated for individuals 10 through 25 years of age for active immunization against N. meningitidis serogroup B.

Source: Pfizer


FDA Advisory Committee Recommends Roche’s Rituximab for Blood Cancers
Genentech, a member of the Roche Group, has received a favorable vote from the US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee recommending the company’s rituximab/hyaluronidase co-formulation as a subcutaneous injection for treating certain blood cancers, including follicular lymphoma, untreated diffuse large B-cell lymphoma (DLBCL), relapsed or refractory low grade or follicular lymphoma, and relapsed or refractory chronic lymphocytic leukemia (CLL).

The new co-formulation includes the same monoclonal antibody as intravenous Rituxan (rituximab), Roche’s anti-cancer drug, and hyaluronidase, a molecule that helps deliver medicine subcutaneously. The FDA is expected to make a decision on approval by June 26, 2017.

This rituximab/hyaluronidase co-formulation has been available in the European Union since 2014 where it is marketed as the subcutaneous formulation of MabThera (rituximab) and is approved in approximately 50 other countries worldwide.

In the US, Rituxan is currently approved as an intravenous formulation for treating follicular non-Hodgkin’s lymphoma, DLBCL, relapsed or refractory low grade or follicular lymphoma, relapsed or refractory CLL. Other indications in the US include rheumatoid arthritis in combination with methotrexate, and granulomatosis with polyangiitis (Wegener’s Granulomatosis) and microscopic polyangiitis in combination with glucocorticoids. The committee’s vote does not affect intravenous Rituxan’s approved uses in the US or in other countries.

Source: Genentech 


FDA Grants Breakthrough Therapy Status for Roche’s Rituxan in New Autoimmune Indication
Genentech, a member of the Roche Group, was granted breakthrough therapy designation status by the US Food and Drug Administration (FDA) for Rituxan (rituximab) for pemphigus vulgaris, a rare, autoimmune, intraepidermal, blistering disease affecting the skin and mucous membranes. Rituxan is Roche’s top-selling drug with 2016 sales of CHF 7.3 billion ($7.3 billion).

The breakthrough therapy designation status was granted based on data from a trial that evaluated Rituxan plus oral corticosteroid (CS) treatment compared to CS as a first-line treatment for moderate to severe pemphigus. FDA breakthrough therapy designation is intended to expedite the development and review of medicines with early evidence of potential clinical benefit in serious diseases and to help ensure that patients receive access to medicines as soon as possible. This latest designation follows the orphan drug designation granted by the FDA to Rituxan in 2015 for treating pemphigus vulgaris.

In the US, Rituxan is approved by the FDA for treating non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, rheumatoid arthritis in combination with methotrexate, and granulomatosis with polyangiitis (Wegener’s Granulomatosis) and microscopic polyangiitis in combination with glucocorticoids.

Source: Genentech


Shire Gains FDA Fast-Track Status for Congenital Disease Drug
Shire has received fast track designation from the US Food and Drug Administration (FDA) for recombinant ADAMTS13 for treating acute episodes of hereditary thrombotic thrombocytopenic purpura, a life-threatening congenital disease caused by a deficiency in the enzyme ADAMTS13. which can cause clotting and associated organ morbidities.

The FDA’s fast track process is designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. However, it does not guarantee that the FDA will ultimately approve the drug or the timing of any such approval.

Source: Shire


FDA Sets PDUFA Date for Valeant’s Glaucoma Drug
The US Food and Drug Administration (FDA) has set a Prescription Drug User Fee Act (PDUFA) date of August 24, 2017 for its decision on a new drug application filed by Valeant Pharmaceuticals International‘s wholly owned subsidiary, Bausch + Lomb, and Nicox, a Sophia Antipolis, France-headquartered ophthalmic research and development company, for latanoprostene bunod ophthalmic solution, 0.024%.

Latanoprostene bunod, a nitric-oxide donating prostaglandin F2α analog for ophthalmic use, is an intraocular pressure lowering single-agent eye drop for treating open angle glaucoma, or ocular hypertension (OHT). It was licensed by Nicox to Bausch + Lomb.

Source: Valeant Pharmaceuticals International

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