Biogen, BMS, Novartis, Novo Nordisk, and Pfizer Lead Drug Approval NewsBy
A roundup of the latest drug approval news, including from the pharmaceutical majors, featuring news from Biogen, Bristol-Myers Squibb, Novartis, Novo Nordisk, and Pfizer
Editor’s Note: This article was updated on a continuous basis for news announced from Wednesday May 31, 2017 to Tuesday June 6, 2017.
EC Approves Biogen’s Antisense Drug Spinraza
The European Commission (EC) has granted a marketing authorization for Biogen’s Spinraza (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA). SMA is a genetic cause of death in infants that is marked by progressive muscle weakness.
Spinraza was first approved by the US Food and Drug Administration in December 2016 within three months of regulatory filing. Biogen has also submitted regulatory filings in Japan, Canada, Australia, Switzerland, and Brazil and plans to initiate additional filings in other countries in 2017.
Biogen licensed the global rights to develop, manufacture, and commercialize Spinraza from Ionis Pharmaceuticals, a pharmaceutical company developing in antisense therapeutics. Biogen and Ionis conducted an innovative clinical development program that moved the drug from its first dose in humans in 2011 to its first regulatory approval in five years. Based on the EC authorization, Ionis will receive a $50-million milestone payment. Ionis is also eligible to receive tiered royalties on global sales of the drug up to a percentage in the mid-teens.
Spinraza is an antisense oligonucleotide (ASO), using Ionis Pharmaceuticals’ proprietary antisense technology, and is designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q that leads to SMN protein deficiency. The drug alters the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein. ASOs are short synthetic strings of nucleotides designed to selectively bind to target RNA and regulate gene expression. Through use of this technology, the drug has the potential to increase the amount of full-length SMN protein in individuals with SMA.
EC Approves BMS’ Immon-Oncology Therapy Opdivo for Bladder Cancer
The European Commission (EC) has approved Bristol-Myers Squibb’s Opdivo (nivolumab) for the treatment of locally advanced unresectable or metastatic urothelial carcinoma,, a common form of bladder cancer, in adults after failure of prior platinum-containing therapy.
Opdivo is Bristol-Myers Squibb’s immuno-oncology drug with 2016 sales of $3.77 billion and is approved for multiple cancer indications.. 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
EC Approves Label Change for Novartis’ Leukemia Drug
The European Commission (EC) has approved the inclusion of Treatment-free Remission (TFR) data in Novartis’ Tasigna (nilotinib) Summary of Product Characteristics. TFR is the ability to maintain molecular response after stopping tyrosine kinase inhibitor therapy in Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) patients in chronic phase..
CML is a type of cancer in which the body produces cancerous white blood cells. Almost all patients with CML have an abnormality known as the Philadelphia chromosome, which produces a protein called BCR-ABL. BCR-ABL causes malignant white blood cells to proliferate. Worldwide, CML is responsible for approximately 10% to 15% of all adult cases of leukemia, with an incidence of one to two cases per 100,000 people per year, according to information from Novartis.
Tasigna (nilotinib) is approved in more than 122 countries for the treatment of chronic phase and accelerated phase Philadelphia chromosome-positive chronic myelogenous leukemia (Ph+ CML) in adult patients resistant or intolerant to at least one prior therapy, including Novartis’ Glivec (imatinib), and in more than 110 countries for the treatment of adult patients with newly diagnosed Ph+ CML in chronic phase.
Novo Nordisk Receives US, European Approval for Hemophilia B Drug
The European Commission has granted marketing authorization for Novo Nordisk’s Refixia (nonacog beta pegol; N9-GP) for the treatment of adolescents and adults with hemophilia B. The authorization covers all 28 European Union member states.
Refixia is indicated for prophylaxis, on-demand treatment of bleeding and surgical procedures in adolescent (greater than 12 years of age) and adult patients with hemophilia B (congenital factor IX deficiency). Novo Nordisk expects to launch Refixia in the first European countries in the fourth quarter of 2017. The US Food and Drug Administration approved the drug under the brand name Rebinyn in May 2017 for on-demand treatment and control of bleeding episodes and the perioperative management of bleeding around the time of surgery in adults and children with hemophilia B.
Refixia (nonacog beta pegol; N9-GP) is an extended half-life factor IX molecule for replacement therapy in patients with hemophilia B. Glycopegylation, the prolongation technology used for the half-life extension, is an approach in hemophilia B. Pegylated products have been approved in hemophilia A and other therapeutic areas.
Source: Novo Nordisk
Pfizer Gets EC Approval for Meningococcal Vaccine
The European Commission (EC) has approved Pfizer’s Trumenba (meningococcal Group B vaccine) for the prevention of invasive meningococcal disease caused by Neisseria meningitidis serogroup B (MenB) in individuals 10 years of age and older.
Adolescents and young adults are a critical demographic for vaccination against MenB due to inherent environmental and social risk factors such as close-quartered living and sharing behavior. The vaccine was first introduced in the United States in October 2014 for active immunization to prevent invasive disease caused by Neisseria meningitidis serogroup B in individuals 10 through 25 years of age.
Pfizer said it continues to in manufacturing processes and facilities to ensure a sufficient supply of Trumenba in Europe, where the majority of meningococcal disease cases (60%) among adolescents and young adults are caused by serogroup B.
Trumenba is a sterile suspension composed of two recombinant lipidated factor H binding protein (fHBP) variants from N. meningitidis serogroup B, one from fHBP subfamily A and one from subfamily B (A05 and B01, respectively). fHBP is one of many proteins found on the surface of meningococci and contributes to the ability of the bacterium to avoid host defenses. fHBPs can be categorized into two immunologically distinct subfamilies, A and B. The susceptibility of serogroup B meningococci to complement-mediated, antibody-dependent killing following vaccination with Trumbena is dependent on both the antigenic similarity of the bacterial and vaccine fHBPs, as well as the amount of fHBP expressed on the surface of the invading meningococci, according to information from Pfizer.