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FDA Breakthrough Therapy Designation: Fifth Drug Receives FDA Approval

DNA Purification

The FDA approves on April 17th, GlaxoSmithKline’s orphan drug Arzerra (Ofatumumab injection for intravenous infusion), the 5th drug to have the coveted Breakthrough Therapy Designation (BTD). A Supplemental Biologic License Application (sBLA) for Arzerra receives approval for use in combination with Chlorambucil, for the treatment of previously untreated patients with Chronic Lymphocytic Leukemia (CLL), for whom fludarabine-based therapy is considered inappropriate. The BTD is for the same indication.

Arzerra is currently marketed in the United States and is granted FDA Accelerated approval in 2009 for CLL refractory to Fludarabine and Alemtuzumab (Campath). As a condition of the 2009 approval, GlaxoSmithKline is required to have further studies. Per the FDA, the trial used for the basis of the April 17th approval fulfilled that 2009 postmarketing requirement, and accelerated approval is converted to regular approval :

FDA Orphan Drug Database Record
Generic Name: Ofatumumab
Trade Name: Arzerra
Date Designated: 03-10-2009
Orphan Designation: Treatment of chronic lymphocytic leukemia
Orphan Designation Status: Designated/Approved
FDA Orphan Approval Status: Approved for Orphan Indication
Approved Labeled Indication: Treatment of chronic lymphocytic leukemia (CLL) refractory to alemtuzumab and fludarabine
Marketing Approval Date: 10-26-2009
Exclusivity End Date: 10-26-2016
Approved Labeled Indication: Ofatumumab in combination with chlorambucil, for the treatment of previously untreated patients with chronic lymphocytic leukemia (CLL) for whom fludarabine-based therapy is considered inappropriate.
Marketing Approval Date: 04-17-2014
Exclusivity End Date: N\A
Sponsor: GlaxoSmithKline One Franklin Plaza Philadelphia, PA 19101 The sponsor address listed is the last reported by the sponsor to OOPD.


Of the 5 BTD approvals, 3 are for the CLL indication:

Row Num Drug Name FDA Approval Date Sponsor Company Indication
1 Gazyva   (Obinutuzumab) 11.01.13 Genentech Chronic Lymphocytic Leukemia (CLL)
2 Imbruvica   (Ibrutinib) 02.12.14 Pharmacyclics Chronic Lymphocytic Leukemia (CLL)
3 Ofatumumab (Arzerra) 04.17.14 GlaxoSmithKline In combination with Chlorambucil for previously untreated Patients with CLL for whom fludarabine-based therapy is considered inappropriate



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “DNA Purification” Courtesy of Dr. Bruce Chassey Laboratory. National Institute Of Dental Research. Li-shan. NCI [Public domain in the US].

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

Orphan Drugs And Rare Diseases: The Heroes Wall


At next week’s World Orphan Drug Congress (WODC) USA, in Washington D.C., one will find the Heroes Wall. The WODC USA has asked advocacy groups and rare disease patients who inspires them. Photos of 30 nominated Heroes of this community and why they are considered heroes, will be displayed at the Heroes Wall. The Heroes Wall will be located in the patient zone, in the exhibition area at the conference.

For an advance look at some of the nominated Heroes, please go to the website.

You may also be interested in participating at the WODC USA, April 23-25, 2014, in Washington DC. WODC USA is where industry, government, and payers come to develop new strategies and partnerships to advance orphan drug development and patient access.

The Orphan Druganaut Blog is a Media Partner of the WODC USA.  We will be attending and look forward to meeting everyone in the orphan drug and rare disease communities. See you there!

Please Note: LuMaxArt Computer Workgroup Concept courtesy of The Gold Guys Blog [CC-BY-SA 2.0] | Wikimedia Commons..

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

Orphan Drugs: 2013 By The Numbers


Two recent 2013 drug trend reports are trending on the Internet. This Blog Post discusses both reports and the 2013 trends in orphan and specialty drugs.

A March Blog Post presents the Medical Marketing & Media (MM&M) article that discusses orphan and specialty drugs and what the future holds for this category of drugs in the United States. Per the article, as orphan and specialty drugs increase over the next few years along with drug prices for these medicines, insurers will need to find a way of minimizing the expense to both patients and to themselves. Also at issue, is the increase in the difficulty of obtaining these medicines for the patient.

Some interesting statistics gathered from the MM&M article:

•   Specialty drugs are the fastest-growing segment of healthcare expenses

•   Specialty drugs are growing at an annual rate of 15% – 20%

•   In 2020, specialty drugs are expected to be 40% of total drug costs

•   In the United States, specialty drug spending is estimated to increase 67% by the end of 2015, while non-specialty drug spending is estimated to decrease 4%.

I – Express Scripts Report

Express Scripts, headquartered in Missouri, is one of the largest Pharmacy Benefit Management (PBM) organizations in the United States. The company provides PBM services such as:

•   Network-pharmacy claims processing

•   Home delivery pharmacy services

•   Specialty PBM

•   Drug formulary management

•   Drug and medical data analysis to manage PBMs for health plans.

Express Scripts publishes annually since 1997, The Drug Trend Report, which provides the “healthcare industry’s most detailed analysis of prescription drug costs and utilization”. The “2013 Drug Trend Report Highlights” is published just this month. The definition and distinction between specialty and orphan drugs is important. In the United States, an orphan drug is defined by the FDA as a drug developed for “a rare disease affecting fewer than 200,000 people”. An orphan drug can be a specialty drug, with specialty drugs focusing “on delivery rather than target.” Specialty drugs usually have complicated usage requirements (i.e. injectable). Specialty drugs often treat chronic conditions such as cancer, hemophilia, and HIV.

Here are a few observations for the drug trends for specialty drugs in 2013 from the report:

•   Drug trends for traditional drugs increase 2.4%, while for specialty drugs there was a 14.1% increase (lowest rate since 2007)

•   Specialty drug spending is 27.7% of total pharmacy benefit spending, up from 24.4% in 2012

•   Specialty drug spending for the top 4 conditions (inflammatory conditions, cancer, multiple sclerosis, and HIV) is 72% of total per-member, per-year (PMPY) spend. Traditional drug spending on the other hand for the top 4 conditions (diabetes, high blood cholesterol, high blood pressure/heart disease, and ulcer disease) is only 34% of total PMPY spend

•   Specialty drug spend is forecast to grow an additional 63%, 2014 – 2016, due to the increase in Hepatitis C (HCV) – note to the reader to keep an eye on the competitive HCV market with 6 FDA Breakthrough Therapy Designations having this indication.

II – IMS Institute For Healthcare Informatics Report

IMS Institute for Healthcare Informatics publishes on April 15th the “Medicine use and shifting costs of healthcare” report. The report reviews in detail the use of drugs in the United States in 2013.

Here are a few observations for the drug trends for orphan drugs in 2013 from the report:

•   Out of 36 New Molecular Entities (NMEs) launched, 17 or approximately 42% are orphan drugs – the most in any year since the Orphan Drug Act is enacted in 1983 & more than double the # launched in 2012

•   In last 5 years, 53 orphan drugs are launched

•   Launches are strengthened by a high # of orphan approvals and increase in applications and approvals for products seeking the Breakthrough Therapy Designation – “signifying a shift towards expediting the availability of drugs to patients in critical need”.

Please Note: “3D Bar Graph Meeting” by “The Gold Guys Blog” [CC-BY-SA-2.0] via Wikimedia Commons.

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

Europe’s April 2014 Products Recommended For Orphan Drug Designation


The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting April 8 – 9, 2014. The EMA COMP April 2014 Meeting Report on the review of applications for orphan designation is published April 14.

At this meeting, there are 12 positive opinions recommending the following medicines for designation as orphan medicinal products. COMP’s opinions are then forwarded to the European Commission (EC). The EC will then decide whether to grant an orphan designation for the medicines in question. Public summaries of the opinions will be available on the EMA website following adoption of the respective decisions on orphan designation by the EC :

 Product Name Sponsor Company Indication
(5R,5aR,8aR,9S)-9-[[4,6-O-[(R)-Ethylidene]-β-D-glucopyranosyl]-oxy]-5-(4-({[(2,2-dimethyl-1,3-dioxolan-4-yl)methoxy]carbonyl}oxy)-3,5-dimethoxyphenyl)-5,8,8a,9-tetrahydroisobenzofuro[5,6-f][1,3]benzodioxol-6(5aH)-one CellAct Pharma GmbH Biliary Tract Cancer
177Lu-tetraxetan-tetulomab Nordic Nanovector AS Follicular Lymphoma
Lutetium (177Lu) edotreotide ITG Isotope Technologies Garching GmbH Gastro-entero-pancreatic neuroendocrine tumors
Recombinant human alpha 1 chain homotrimer of type VII collagen Shire Pharmaceuticals (Ireland) Epidermolysis Bullosa
4-(4-Methoxy-phenylamino)-6-methylcarbamyl-quinoline-3-carboxylic acid Clanotech AB Prevention of scarring in post glaucoma filtration surgery
Adeno-associated viral vector serotype 2 containing the human CHM gene encoding human Rab escort protein 1 Alan Boyd Consultants Ltd Choroideraemia
Aganirsen Gene Signal SAS Central retinal vein occlusion
Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene Cochamo Systems Ltd Mucopolysaccharidosis IIIA (Sanfilippo A syndrome) 
Autologous dendritic cells pulsed with RNA from glioma stem cells Epitarget AS Glioma
Isavuconazonium sulfate Astellas Pharma Europe Mucormycosis 
Paclitaxel-succinate-Arg-Arg-Leu-Ser-Tyr-Ser-Arg-Arg-Arg-Phe CLL Pharma Glioma
Plasmid DNA encoding the human cystic fibrosis transmembrane conductance regulator gene complexed with a non-viral,cationic lipid based gene transfer agent Imperial Innovations Limited Cystic Fibrosis


Please Note: “Erlenmeyer Flasks” From Argonne US National Lab  [Public domain in the US] | Wikimedia Commons.

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

Social Media And Patients: SmartPhone Apps

Global Internet

This is the second Blog Post in a series that examines the use of different social media channels by patients and why from an emotional perspective it is used by so many patients with chronic and rare diseases.

The use of social media tools, smartphone apps, and online communities are used by pharmaceutical companies and patient advocacy groups to reach out and provide support to patients and their families affected by rare or orphan diseases. The purpose of these programs is to help the rare disease community connect with others with rare diseases to help eliminate the aloneness the patient may be feeling. Other purposes of smartphone apps for the rare disease community :

•   Provide a mechanism for monitoring and tracking a disease

•   Provide remote diagnostics

•   Information on disease

•   Communication with medical support team.

According to the Pew Internet & American Life Project Mobile Health Report :

•   85% of American adults own a cell phone, with 53% of those owning smartphones

•   52% of smartphone owners gather health information on their phones

•   19% of smartphone owners have at least one health app on their phone

I – Epidermolysis Bullosa (EB)

Epidermolysis Bullosa (EB) is a rare genetic connective tissue condition that involves very fragile skin that blisters or tears with the slightest trauma or friction. EB patients are known as “Butterfly children” due to the fragile nature of the skin, similar to the fragility of the wings of a butterfly. There is currently no cure or effective treatment, outside of daily wound care, pain management, and preventative bandaging.

The patient advocacy group DEBRA International, in November 2013, announces a new smartphone app, iscorEB (instrument for Scoring Clinical Outcomes for Research of EB), to be used for scoring the severity of a patient’s EB. iscorEB is developed by The Hospital for Sick Children (SickKids) in Toronto, Canada. iscorEB quantifies the severity of EB from both the patient/family and clinician perspective – used to monitor EB progression in clinical practice or by the patient themselves to measure changes over time. iscorEB is abailable FREE of charge from the iTunes Store and will be available soon on Google play stores.

II – Hemophilia

There are many Hemophilia apps currently available with the purpose of helping patients track their hemophilia healthcare information.

BioRx, a specialty pharmacy and infusion services provider located in Ohio, announces in October 2013, the launch of a new app, MyFactor. The purpose of MyFactor is to give both patients and caregivers a place to monitor and manage home treatment for Hemophilia and related bleeding disorders. The app provides the ability to record, store, view, and transmit medical information related to a deficiency of clotting Factor VII, VIII, IX, XIII, or von Willebrand factor. MyFactor is available on iTunes. A release for Android devices is planned for 2014.

Please Note: “Applications-internet” by The Tango!Desktop Project | Wikimedia Commons.

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

FDA Breakthrough Therapy Designation: #37 From CBER For Gene Therapy


On April 10th, Celladon Corporation, a San Diego-based biotechnology company, announces that the FDA ‘s Center for Biologics Evaluation and Research (CBER) grants the company’s lead candidate, Mydicar, a Breakthrough Therapy Designation (BTD). The BTD is granted to Mydicar for reducing hospitalizations for Heart Failure (HF) in NYHA class III or IV chronic HF patients who are NAb negative. Mydicar is a novel, 1st-in-class therapy for chronic HF due to systolic dysfunction. Mydicar uses gene therapy to :

“ .. selectively target and restore SERCA2a enzyme levels by transferring the SERCA2a gene directly into cardiac muscle cells, which improves the heart’s ability to pump blood … uses a non-pathogenic recombinant adeno-associated virus (AAV) and is delivered directly to the heart in a routine outpatient procedure .. “.

Also, Celladon Corporation has developed a companion diagnostic to determine patients who are AAV1 NAb negative and thus are eligible for Mydicar. The company says that there are about 350,000 US HF patients who may be eligible.

Mydicar is currently in a Phase IIb CUPID 2 trial, with results expected in April 2015.

This is the 37th BTD announced by a sponsor company, the 6th BTD for 2014, and :

“… the first gene therapy reported to receive BTD from FDA’s CBER ….”.

CBER is the FDA’s Center for Biologics Evaluation and Research that regulates biological products for human use, such as allergenics, blood, devices, gene therapy, human tissues and cellular products, vaccines, and xenotransplantation products. As of 03/31/14 (reference latest BTD Statistics Chart), there are 2 CBER BTDs.

In Europe, the EMA (European Medicines Agency), has given regulatory approval to uniQure, to sell its gene therapy, Glybera, for the treatment of the rare disease, Lipoprotein Lipase Deficiency (LPLD). No gene therapy has ever been approved by the FDA.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “Glowing heart (114668824)” by 29cm from Hong Kong (Glowing heart) [CC-BY-SA-2.0] | Wikimedia Commons..

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

Social Media And Patients: Patient-To-Patient Blogs

Global Internet

This is the first Blog Post in a series that examines the use of different social media channels by patients and why from an emotional perspective it is used by so many patients with chronic and rare diseases.

Online patient Blogs provide opportunities to make global connections with others experiencing the same challenges and ups and downs with one’s disease or illness. Blogs that are created and maintained by rare disease patients can be a vital source of emotional support, a forum for helpful discussion and sharing of resources, information and diagnostics, sharing of personal experiences with different drugs and treatments, and just providing a spot where the patient can communicate with others in the same situation. Blogs become a “safe space” for rare disease patients to share. There can be positive shares, but there can also be times of confusion, pain, grief, and sadness. Blogs are a place where global stories can be expressed and heard. Blogging for a rare disease patient can increase empowerment, communication and social support, and helps one not feel alone but part of a community where everyone understands what one is experiencing and going through. Blogs can be a “safe have” for the rare disease patient.

Blogs can also give medical and healthcare professionals a glimpse into the world of a rare disease patient, help better understand the world of having a rare disease. The blogosphere is a place for providers to understand and become informed from a patients’ story or narratives. It is a means for the provider to observe from afar how the rare disease is impacting the patient and their family on a day by day basis. It also lets the provider and outside world know what it is like to go through the medical system in a particular country. The Blog becomes the diary of a rare disease patient. The Blog becomes a tool that can help close the communication gap between patient, doctors, and healthcare professionals.

Please Note: “Applications-internet” by The Tango!Desktop Project | Wikimedia Commons.

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


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