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FDA GRANTS PRIORITY REVIEW TO GENENTECH’S HEMLIBRA FOR PEOPLE WITH HEMOPHILIA A WITHOUT FACTOR VIII INHIBITORS

June 5, 2018

The following is an excerpt from a press release from Genentech. Read the entire press release here. Genentech, a member of the Roche Group, announced the U.S. Food and Drug Administration has accepted the company’s supplemental Biologics License Application and granted Priority Review for HEMLIBRA  for adults and children with hemophilia A without factor VIII […]

Bioverativ Highlights the Impact of Humanitarian Aid in Hemophilia

April 17, 2018

The following is an excerpt from a press release from Bioverativ. Read the entire press release here.  More than 15,000 people with hemophilia in 40 developing countries have already been treated following Bioverativ and Sobi’s unprecedented donation of factor therapy to the WFH Humanitarian Aid Program Bioverativ Inc., a Sanofi company, joins the global hemophilia […]

FDA Grants Breakthrough Therapy Designation for Genentech’s Hemlibra

April 17, 2018

The following is an excerpt from a press release from Genentech. Read the full press release here.  Genentech, a member of the Roche Group, announced today the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to HEMLIBRA for people with hemophilia A without factor VIII inhibitors. Breakthrough Therapy Designation is designed to accelerate the […]

FDA Advises CSL Behring Their Promotional Materials are Misleading

March 6, 2018

Note: The following is a summary of a letter dated Feb. 27, 2018, from the US Food and Drug Administration (FDA) to CSL Behring. You can read the original letter here. The FDA has advised CSL Behring that the company’s promotional materials make misleading claims about the effectiveness of its Idelvion (Factor IX) product. “Such […]

FDA Lifts Clinical Hold on Fitusiran

December 15, 2017

Note: The following is edited from a press release from Alnylam Pharmaceuticals. Read the full press release in its entirety here. Alnylam Pharmaceuticals Inc., a RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced Dec. 15, 2017, the U.S. Food and Drug Administration (FDA) has lifted the hold on clinical studies with fitusiran, including […]

Bayer Issues Voluntary Recall of Kogenate FS

October 18, 2017

Note: The following is the full text of an email communication received by HFA from Bayer HealthCare Pharmaceuticals: Bayer in consultation with the FDA has voluntarily recalled approximately 300 vials (part of one lot) of Kogenate® FS, Antihemophilic Factor (Recombinant) in the U.S. as an excipient, used to stabilize the product during manufacturing, may not conform […]

Shire’s Adynovate Receives Two New FDA Approvals for Treatment of Hemophilia A

December 29, 2016

Note: The following is edited from a press release from Shire. Read the full press release in it’s entirety here. Shire plc recently announced that the U.S. Food and Drug Administration (FDA) has approved Adynovate, an extended circulating half-life recombinant Factor VIII treatment for hemophilia A, in pediatric patients under 12 years of age. The FDA […]

FDA Issues Warning New Hepatitis C Drugs Could Reactivate Hepatitis B Virus

October 9, 2016

The following is excerpted from an FDA news release. To read the release in its entirety, click here. The U.S. Food and Drug Administration is warning about the risk that some of the new direct-acting antiviral medicines for hepatitis C (HCV) can reactivate the hepatitis B virus (HBV) among patients who have had HBV. “In a […]

Update #2: Zika Virus & Blood Supply

August 10, 2016

For the past few months, HFA has been monitoring the progression of the Zika virus in the United States. Since the first reported cases of Zika virus reaching the United States in January, there have been thousands of additional cases. According to the Centers for Disease Control (CDC), the follow cases and causes of transmission […]

FDA Requests Comments Regarding MSM Donor Deferral Policy

August 10, 2016

In January of this year, the Food and Drug Administration (FDA) revised its donor deferral policy surrounding men who have sex with men (MSM). On July 26, 2016, the FDA issued another request for comments regarding the MSM donor deferral policy. Current policy permits blood donations by MSM provided that the donor has not had […]

Spark Therapeutics and Pfizer Announce Receipt of FDA Breakthrough Therapy Designation for SPK-9001 for the Treatment of Hemophilia B

July 21, 2016

Note: The below is an edited version of a press release by Spark Therapeutics. The original release can be read in it’s entirety here. Spark Therapeutics and Pfizer Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to SPK-9001, the lead investigational candidate in the companies’ SPK-FIX program, in development for the treatment of hemophilia B. SPK-9001, a […]

FDA Releases “Voice of the Patient” Summary from Patient-Focused Drug Development Initiative

July 14, 2016

In September 2014, the U.S. Food and Drug Administration hosted a public meeting as part of it’s Patient-Focused Drug Development initiative, a 5 year initiative to learn more from patients across a variety of disease and disorders. We were proud to be a part of this meeting and provided support to several of the patient […]

FDA Approves First Single Chain Product for Hemophilia A

May 27, 2016

Note: The below is an edited version of a press release by CSL Behring. The original release can be read in it’s entirety here. KING OF PRUSSIA, Pa., May 26, 2016 — CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has approved AFSTYLA [Antihemophilic Factor (Recombinant), Single Chain], its novel long-lasting recombinant factor […]

Letter to FDA Commissioner Regarding Biosimiliar Safety & Approval Process

April 20, 2016

HFA is proud to be part of the Patients for Biologics Safety & Access (PBSA). PBSA is a coalition of 24 patient advocacy organizations dedicated to protecting patient access to safe and effective biologics. Together, this coalition represents millions of Americans who suffer from serious, life-threatening diseases that are difficult to diagnose and treat. As […]

FDA Approves Treatment of Chronic Hepatitis C Genotypes 1 and 4

February 1, 2016

Note: The following is an edited form of a press release from the Food and Drug Administration. The original form of the release can be read here. On Thursday, January 28, the U.S. Food and Drug Administration approved Zepatier (elbasvir and grazoprevir) with or without ribavirin for the treatment of chronic hepatitis C virus (HCV) […]

FDA’s Completed Revision of MSM Donor Deferral Policy

January 28, 2016

Background The Food & Drug Administration (FDA) is responsible for recommending policies and making rules regarding the donation of blood and blood products. During the 1980s, when it became clear that transmission of Acquired Immune Deficiency Syndrome (AIDS) happened through blood, the FDA began recommending multiple policies that would decrease the risk of transmission of […]

FDA Approves Twice-Weekly Hemophilia A Treatment

November 16, 2015

Note: The following is an edited version of a press release originally published by Baxalta. The original release can be read here. Baxalta Incorporated announced on Monday, November 16, 2015, that the U.S. Food and Drug Administration (FDA) has approved ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated], an extended circulating half-life recombinant Factor VIII (rFVIII) treatment for hemophilia […]

FDA Approves First Factor X Concentrate for Treatment of a Rare Hereditary Bleeding Disorder

October 21, 2015

Note: The following is an edited version of a press release originally published by the US Food and Drug Administration (FDA). The original release can be read here. The U.S. Food and Drug Administration announced on Tuesday, October 20, 2015, the approval of  Coagadex, Coagulation Factor X (Human), for hereditary Factor X (10) deficiency. Until […]

FDA Approves Fast Track Designation for New Hemophilia B Treatment

September 18, 2015

Note: This is an edited form of a press release from Dimension Therapeutics, Inc. To read the original release in its entirety, click here. Dimension Therapeutics, Inc. announced on Thursday, September 17 that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the company’s lead product candidate, DTX101, for the treatment of […]

FDA Clears Two New Oral Drugs for Hepatitis C

July 27, 2015

Note: This article originally appeared on Medscape. To read the FDA release on Daklinza (genotype 3), click here. For the FDA release regarding Technivie (genotype 4), click here. The US Food and Drug Administration (FDA) approved two new drugs today for hepatitis C (HCV), one for genotype 3 and the other for genotype 4. The first medication is […]

FDA Grants Orphan Status for New Inhibitor Treatment

July 2, 2015

Below is from a press release Apitope sent out on June 9, 2015. To read it in its entirety, click here. Apitope, the drug discovery and development company focused on disease-modifying treatments that reinstate immune tolerance, announced today that pre-clinical product candidate ATX-F8-117 has been granted Orphan Drug Status by the US Food and Drug […]

FDA Making Progress With Blood Safety Monitoring System

May 15, 2015

Last December, Food and Drug Administration (FDA) Commissioner Margaret A. Hamburg  set forth a recommendation to change the current blood donation policy for men who have sex with men (MSM). The old policy, set forth in the height of the AIDS crisis of the 1980s, barred men who have had sex with men since 1977 from donating blood. […]

FDA Approves New Hemophilia B Treatment

April 30, 2015

  Note: This is an edited version of a Emergent BioSolutions Inc. press release. To read the full release, click here. Emergent BioSolutions Inc. announced on Thursday, April 29 the U.S. Food and Drug Administration (FDA) has approved IXINITY®, an intravenous recombinant human coagulation factor IX therapeutic for the control and prevention of bleeding episodes and for perioperative management […]

FDA To Review CSL Behring’s New Long-Lasting Hemophilia B Product

February 4, 2015

CSL Behring announced that the U.S. Food and Drug Administration (FDA) has accepted for review its Biologics License Application (BLA) for the marketing authorization of its long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP). Upon FDA approval, rIX-FP will provide hemophilia B patients with a long-acting treatment option with dosing intervals up […]

FDA approves Viekira Pak to treat Hepatitis C

January 5, 2015

The U.S. Food and Drug Administration today approved Viekira Pak (ombitasvir, paritaprevir and ritonavir tablets co-packaged with dasabuvir tablets) to treat patients with chronic hepatitis C virus (HCV) genotype 1 infection, including those with a type of advanced liver disease called cirrhosis. Hepatitis C is a viral disease that causes inflammation of the liver that […]

FDA Makes Statement on MSM Blood Donor Deferral Policy

December 23, 2014

Today, Commissioner Margaret A. Hamburg of the Food and Drug Administration (FDA) set forth a recommendation to change the current MSM blood donation policy. The current policy, set forth in the height of the AIDS crisis of the 1980s, forbade men who have had sex with men since 1977 from donating blood. The new policy […]

Baxter Files For FDA Approval of Recombinant Treatment For von Willebrand Disease

December 22, 2014

Baxter International Inc. today announced that the company has submitted a biologics license application (BLA) to the United States (U.S.) Food and Drug Administration (FDA) for the approval of BAX111, the first highly-purified recombinant von Willebrand Factor (rVWF) in clinical development as a treatment for patients with von Willebrand disease, the most common type of […]

FDA Approves New Treatment for Rare Form of Hemophilia

October 25, 2014

On Friday, October 24, 2014, the U.S. Food and Drug Administration approved Obizur [Antihemophilic Factor (Recombinant), Porcine Sequence] for the treatment of bleeding episodes in adults with acquired hemophilia A (acquired Factor VIII [FVIII] deficiency). Acquired hemophilia A is a rare, but potentially life threatening, bleeding disorder caused by the development of antibodies (immune system […]

FDA Approves First Combination Pill to Treat Hepatitis C

October 10, 2014

The U.S. Food and Drug Administration today approved Harvoni (ledipasvir and sofosbuvir) to treat chronic hepatitis C virus (HCV) genotype 1 infection. Harvoni is the first combination pill approved to treat chronic HCV genotype 1 infection. It is also the first approved regimen that does not require administration with interferon or ribavirin, two FDA-approved drugs […]

FDA Hosts Patient-Focused Meeting

September 23, 2014

  On Monday, September 22, several members of the bleeding disorders community came out to share their experiences with bleeding disorders treatment with the FDA.* HFA worked with partners like NORD, PPTA, and NHF to ensure that our community was effectively represented. Patients and advocates of every age, every background, and with several different conditions came out […]


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