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FDA Breakthrough Therapy Designation: Pfizer’s Meningococcal Group B Vaccine Gets Accelerated Approval


Pfizer announces on October 29th, that the FDA grants Accelerated Approval of Trumenba (Meningococcal Group B vaccine) for immunization for people, ages 10 – 25, to prevent invasive disease caused by Neisseria meningitidis serogroup B. Trumenba is to be administered as a 3-dose series at months 0, 2, and 6 in the 10 – 25 year old age group.

Trumenba is the 1st and only FDA approved vaccine in the United States for the prevention of Meningococcal Meningitis B. Trumenba is reviewed and approved under the FDA’s Breakthrough Therapy Designation (BTD) and Priority Review programs. Pfizer receives the FDA BTD in March 2014. Part of the FDA Accelerated Approval process requires Pfizer to conduct further studies to verify Trumenba’s effectiveness against additional strains of N. meningitidis serogroup B.

There are five meningococcal serogroups: A,B,C, W-135, and Y. Serogroup B is the only one for which no broadly-protective vaccine is currently approved in the US. Two existing vaccines, Menactra and Menveo, prevent the other four types of bacterial meningitis and are currently recommended by the CDC for all preteens and adolescents. Many schools and colleges require students to get a meningitis vaccine before starting classes. According to the CDC, in the US in 2012, there were about 500 cases of Meningococcal Disease, with 160 cases or approximately 32%, caused by serogroup B. The CDC said that 10 – 15% of these cases are fatal and that for those who survive, 11 – 19% suffer permanent disabilities.

Pfizer’s competitor for this indication in the US is Novartis’s Bexsero Vaccine that receives the FDA BTD in April 2014 Bexsero is not yet approved of in the US. In Europe, Australia, and Canada, Bexsero is already approved for Meningococcal disease caused by serogroup B. In the UK, the Joint Committee on Vaccination and Immunization (JCVI)  recommended in March 2014 including Bexsero in the country’s National Immunization Program (NIP) for “routine use in infants from 2 months of age.”


FDA October 29, 2014 News Release


FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “Flickr – Nicholas T – Bows” by Nicholas A. Tonelli from Pennsylvania, USA (Bows) [CC-BY-2.0] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

Patient Advocacy: Rare Disease Groups’ Use Of Social Media





PatientView, is a UK-based research, publishing, and consultancy group, created out of a belief that “the view of patients should be considered in all important healthcare decisions … formed in response to the emerging powerful new global patient movement.”

PatientView is publishing a series of reports benchmarking the patient movement. Survey data is collected from 1,000 patient groups in 60 countries. The data examines the following 11 therapeutic areas:

•   Cancer
•   Circulatory
•   Diabetes
•   Endocrine
•   Gastrointestinal
•   Mental Health
•   Neurological
•   Rare Disease
•   Respiratory
•   Rheumatology.

One survey question asked whether the patient organization has any of the following social media accounts: website, blog, Twitter account, Facebook account, and other social media tools. The following is observed, per the Report for 2014, for the “rare disease” patient groups:

•   96% have a website

•   75% have a Facebook account

•   36% have a Twitter account

•   18% have a Blog.

The main take-away from this survey question is that social media has become the main means by which patient groups talk to their patient members. The most active therapeutic patient groups in the use of social media, are those that don’t get much media exposure. It is these therapeutic patient groups that “compensate by being active on Facebook and Twitter.” It is the “diabetes” therapeutic patient groups (per the survey) that rank last at the use of social media tools.

Please Note: “Kid Hugging a Rainbow” by Marendo Müller, artwork (Own work) [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

EveryLife Foundation For Rare Diseases: Annual Rare Artist Contest


The EveryLife Foundation for Rare Diseases is “dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy.”  The organization works to improve the clinical development process for treatments for rare diseases by collaborating with multiple stakeholders:

•   Academic scientists
•   Patient organizations
•   FDA
•   Biotechnology/Pharmaceutical Industry
•   National Institutes of Health (NIH).

The EveryLife Foundation for Rare Diseases recently announces the 6th Annual Rare Artist Contest. The contest opened September 30, 2014, for submissions, entry deadline is January 16, 2015, voting until January 23, 2015, and the contest awards to be announced by February 15, 2015.

The EveryLife Art Contest is established in 2010 for artists affected by a rare disease with the purpose of raising awareness of rare diseases and to encourage the telling of their stories using art. is an online gallery that is a free, permanent platform for public viewing. The mission of the contest is:

“ … to showcase the artwork and the Artist, in order to raise awareness about rare diseases and showcase our vibrant community. Art is the expressive medium used to capture the trials and triumphs of our human existence. For rare disease patients, life can be a day-to-day struggle wrought by a devastating disease and unanswerable questions. Art as an expression of these challenges in life and in living each day, can be a particularly powerful medium to guide all of us, in our struggles and successes. Artists affected by rare disease communicate their pain, frustration, optimism and joy, and through their work we can learn more about ourselves … “.

For detail information about the contest and its rules, please reference the website. The contest is being hosted on Facebook.

Please Note: “Jakob Alt – View from the Artist’s Studio in Alservorstadt toward Dornbach, 1836 – Google Art Project” Jakob Alt [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

Rare Disease Start-Up: Dimension Therapeutics’ Gene Therapy For Hemophilia A


This Blog Post reviews the new start-up, Dimension Therapeutics and the company’s first gene therapy program for Hemophilia A.

The creation of Dimension Therapeutics (DT), a new Cambridge MA-based start-up gene therapy company, focusing on the development of treatments for rare diseases, is announced in October 2013. Fidelity Biosciences and REGENX Bioscience announce the creation of DT. The new start-up company will develop and commercialize novel Adeno-Associated Virus (AAV) gene therapy products for multiple rare disease indications.  During gene therapy, an AAV delivers a working “healthy” gene into the body to replace the faulty or missing targeted gene.

Fidelity Biosciences provides the initial venture capital for the new start-up. REGENX Biosciences is an AAV gene therapy company that is providing its proprietary NAV vector technology platform. DT acquires access to REGENX Biosciences’ NAV vector technology and rights to the company’s product programs in multiple rare disease indications. Dimension has now raised $30M with funding from Fidelity Biosciences and OrbiMed.

DT’s lead gene therapy program will be for Hemophilia A. Hemophilia is a rare bleeding disorder that is usually inherited and most often occurs in males. Hemophilia is characterized by producing little or no clotting factor. The clotting factor is a protein required for normal blood clotting. When a hemophilic patient has an injury, a life threatening bleeding can occur. The two types of Hemophilia are Hemophilia A (Factor VIII Deficiency), the most common type, and Hemophilia B (Factor IX).

The main treatment option for Hemophilia is replacement therapy – concentrates of clotting factor are administered intravenously to help replace the clotting factor that is either low or missing in the patient. Gene therapy for Hemophilia corrects the faulty gene that causes it. DT’s AAV vector technology allows the “administration of the gene therapy in vivo, which has been shown in previous proof-of-concept studies in hemophilia to trigger the liver to create the clotting factor. This approach may enable patients to live without replacement therapy infusions for an extended period of time.”

In June 2014, DT and Bayer HealthCare announce a collaboration to develop and commercialize gene therapy for the treatment of Hemophilia A. DT will receive an upfront payment of $20 million and will be eligible for potential milestone payments of up to $232 million. DT is responsible for pre-clinical development and the Phase I/IIa clinical trial, with funding from Bayer. Depending on results of the Phase I/IIa clinical trial, Bayer will conduct the confirmatory Phase III trial, will do the regulatory submissions, and will have worldwide rights to commercialize the future potential product.

In September 2014, FierceBiotech, names DT as one of 2014’s “Fierce 15 biotechnology companies”.

Please Note: “Gene Therapy” National Institute of Health [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

FDA Breakthrough Therapy: Merck’s Keytruda Receives 2nd Designation For Advanced Non-Small Cell Lung Cancer


On October 27th, Merck announces that the FDA Breakthrough Therapy Designation (BTD) is given to Keytruda (Pembrolizumab) for the treatment of Epidermal Growth Factor Receptor (EGFR) mutation-negative, and Anaplastic Lymphoma Kinase (ALK) rearrangement-negative Non-Small Cell Lung Cancer (NSCLC), where the disease progresses on or following platinum-based chemotherapy.

Keytruda is a “humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2. By binding to the PD-1 receptor and blocking the interaction with the receptor ligands, Keytruda releases the PD-1 pathway-mediated inhibition of the immune response, including the anti-tumor immune response.”

The FDA BTD is based on data from the ongoing Phase Ib KEYNOYE-001 study. There are other clinical trials for Keytruda in advanced NSCLC:

•   Ongoing Phase II (KEYNOTE-010)

•   Ongoing Phase III (KEYNOTE-024)

•   Phase III planned to begin 4th quarter 2014 (KEYNOTE-042).

This is the 3rd FDA BTD for Merck and the 2nd FDA BTD for Keytruda (recently approved by the FDA for Melanoma):

 # Drug Name Indication
1 Keytruda (Pembrolizumab) Melanoma
2 Keytruda (Pembrolizumab) EGFR-Negative & ALK Rearrangement-Negative NSCLC




FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “IMG Carlingford Lough 0617” by Sarah777 at en.wikipedia [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

Vaccines: OncoPep’s FDA Orphan Drug Designation For Multiple Myeloma


This is the second Blog Post in a series that reviews FDA Orphan Drug Designations (ODDs) for vaccines.

OncoPep, founded in 2010, is developing targeted immunotherapeutics for oncology indications. OncoPep’s proprietary core technology, licensed from the Dana Farber Cancer Institute, uses:

“ … a unique combination of proprietary peptides to form a therapeutic cancer vaccine. Vaccines developed from this technology are designed to stimulate the patient’s immune system to attack his or her cancer through an optimized combination of disease-specific peptides and adjuvants (substances that stimulate the immune system).

OncoPep’s lead investigational candidate, PVX-410, is a 4-peptide cancer vaccine designed to target specific antigens found on the surface of Multiple Myeloma (MM) cancer cells. PVX-410 receives FDA Orphan Drug Designation (ODD) in June 2013 for this indication. The company is initially developing PVX-410 for the treatment of Smoldering MM (SMM), a precursor to MM.

MM is a blood cancer that affects plasma cells. It is the second most common blood cancer:

•   13% of all hematologic cancers
•     1% of all cancer deaths
•    Median survival is 7-8 years
•    Currently no cure
•    In 2013,about 22,350 new cases were diagnosed (American Cancer Society).

OncoPep is financed by angel groups, individuals, family foundations, and the Leukemia & Lymphoma Society (LLS). In August 2014, OncoPep raises $6.9 million Series B financing to advance novel cancer vaccines and to expand the Phase I/IIa PVX-410 clinical trial  for SMM.

FDA ODD Database For OncoPep’s MM Vaccine

Generic Name: Multi-peptide cancer vaccine
Trade Name: n/a
Date Designated: 06-23-2013
Orphan Designation: Multiple Myeloma
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: OncoPep, Inc. 520 Boston Street North Andover, MA 01845



International Myeloma Foundation (IMF)

Leukemia & Lymphoma Society (LLS)

Multiple Myeloma Research Foundation (MMRF)

The Myeloma Beacon.

Please Note: “Vaccine” by John Keith (Photographer) [Public domain, NIH], via Wikimedia Commons | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

Australia: Orphan Drugs Kalydeco And Soliris Triumph




Australia’s Health Minister Peter Dutton announces on October 26th that the following 2 orphan drugs are to be listed on the Pharmaceutical Benefits Scheme (PBS) effective December 1, 2014:

•   Vertex Pharmaceuticals’ Kalydeco (Ivacaftor) for Cystic Fibrosis (CF)

•   Alexion Pharmaceuticals’ Soliris (Eculizumab) for Atypical Hemolytic Uremic Syndrome (aHUS).

I – Australia’s PBAC & PBS

Australia’s Pharmaceutical Benefits Advisory Committee (PBAC) makes national funding decisions for the Australian public healthcare system.  The PBAC assesses orphan drugs separately from non-orphan drugs using very strict criteria. Australian approval and reimbursement of a new medicine is a multi-step process:

•   Therapeutic Goods Administration (TGA) approves the new medicine

•   Pharmaceutical Benefits Advisory Committee (PBAC) assesses the new medicine for effectiveness and cost-effectiveness

•   Listing on the Pharmaceutical Benefits Scheme (PBS) based on the PBAC assessment.

II – Kalydeco

 The addition of Kalydeco to the PBS follows over a year of discussion between patients, clinicians, Vertex Pharmaceuticals, the Australian government, and the Department of Health. The PBAC defers making a decision on Kalydeco at its July 2013 meeting, recommends Kalydeco in November 2013, and then recommends it for a second time in March 2014. According to Mr. Dutton’s October 26th Press Release:

“ .. The PBS subsidy of this medicine, which would otherwise cost approximately $300,000 a year per patient, will bring great relief to the patients and the families of people affected by this life threatening condition …Consistent with the advice of the Pharmaceutical Benefits Advisory Committee (PBAC) that this medicine could be listed on the PBS based on a pay-for-performance basis, all cystic fibrosis patients six years and older who have a G551D mutation in the CFTR gene will be treated with ivacaftor for as long as needed. I’m pleased to announce that the sponsor of ivacaftor, Vertex Pharmaceuticals, has agreed to proceed with the PBS listing as recommended by the PBAC

 The Australian government approves$174.5 million over the next 4 years to fund Kalydeco on the PBS. More than 250 Australians are affected by the CF G551D gene mutation.

Kalydeco is approved in Australia in July 2013 for patients with at least one copy of the G551D mutation.

III – Soliris & Other Medicines

Soliris (Eculizumab) for the treatment of the ultra-rare disease, Atypical Haemolytic Uraemic (aHUS), will also be added to the PBS effective December 1st of this year. This will help patients and their families meet the $500,000 annual cost per patient per year. The government approves $63 million over the next 4 years to provide Soliris on the PBS.

Nine other new and amended medicines are to be listed on the PBS starting December 1, 2014, including treatments for Hepatitis C and prostate cancer. All PBS listings are subject to final arrangements being met by the suppliers/sponsor companies of the medicines.


CF Australia’s Statement

Minister Dutton’s Statement

Vertex Pharmaceuticals’ Statement.

Please Note: “Chemlab2” by Tahoenathan (Own work) [CC-BY-SA-3.0] | via Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.


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