美国和欧盟的药品监管机构均接受了百健艾迪旗下长效多发性硬化症药物Plegridy的上市申请，使得这款药物可能会于2014年上半年获得批准。

Plegridy（聚乙二醇干扰素β-1a）是百健艾迪旗下重磅炸弹级多发性硬化症药物Avonex（干扰素β-1a）的一种新制剂，Avonex目前还是该公司销售最好的产品，其2012年的销售额达到29.1亿美元。百健艾迪曾希望Plegridy能够获得FDA优先审评权，但事与愿违，FDA计划以标准审评程序对这款药物进行审评，审评期限大约10个月。

Plegridy的申报资料基于ADVANCE III期临床研究第一年的试验结果，结果显示Plegridy与安慰剂相比，能明显降低多发性硬化症患者的疾病活动情况，减少复发次数及减缓残疾进展和脑部病变。临床研究中，这款药物也有良好的安全性和耐受性。

Plegridy的受益是其与Avonex相比需要注射的频次较少，并且Plegridy以皮下注射而非肌肉注射，所以使用时较少有疼痛感。此外，Plegridy每两周或四周才注射一次，而Avonex需要每周注射一次。

去年，随着Avonex一种新型笔式注射器在美国和欧盟市场的铺开，百健艾迪已经获准延长了这款药物的专营权，但Plegridy有更加明显的发展优势，因为它是一种新的分子实体(NME)，因而可以延长公司干扰素β的专利寿命和专营权。

Avonex的专利于今年年底到期，Plegridy的获批将会帮助百健艾迪保持其干扰素的专营权，另外，该公司其它多发性硬化症药物也在成长，包括口服药物Tecfidera（富马酸二甲酯），这款药物在未来几年可能会主导多发性硬化症药物市场，其销售峰值每年将会超过30亿美元。百健艾迪也在销售多发性硬化症药物Tysabri（那他珠单抗），这款药物对某些患者来说仍是一个重要的治疗选择，此外，百健艾迪与Idec合作开发一种新型抗-CD-25抗体正处于后期临床研究阶段。


 

Plegridy
(peginterferon beta-1a) prefilled syringe and Plegridy Pen (peginterferon beta-1a) prefilled pen
Company:  Biogen Idec, Inc.
Application No.:  125499
Approval Date: 8/15/2014

Persons with disabilities having problems accessing the PDF files below may call (301) 796-3634 for assistance.

• Approval Letter(s) (PDF)
• Printed Labeling (PDF)
• Summary Review (PDF)
• Officer/Employee List (PDF)
• Office Director Memo (PDF)
• Cross Discipline Team Leader Review (PDF)
• Medical Review(s) (PDF)
• Chemistry Review(s) (PDF)
• Pharmacology Review(s) (PDF)
• Statistical Review(s) (PDF)
• Microbiology Review(s) (PDF)
• Clinical Pharmacology Biopharmaceutics Review(s) (PDF)
• Risk Assessment and Risk Mitigation Review(s) (PDF)
• Proprietary Name Review(s) (PDF)
• Other Review(s) (PDF)
• Administrative Document(s) & Correspondence (PDF)

Important Safety Information

PLEGRIDY can cause serious side effects. Call your healthcare provider right away if you have any of the symptoms listed below.

• Liver problems, or worsening of liver problems including liver failure and death. Symptoms may include yellowing of your skin or the white part of your eye, nausea, loss of appetite, tiredness, bleeding more easily than normal, confusion, sleepiness, dark colored urine, and pale stools. During your treatment with PLEGRIDY you will need to see your healthcare provider regularly. You will have regular blood tests to check for these possible side effects
• Depression or suicidal thoughts. Symptoms may include new or worsening depression (feeling hopeless or bad about yourself), thoughts of hurting yourself or suicide, irritability (getting upset easily), nervousness, or new or worsening anxiety

Do not take PLEGRIDY if you are allergic to interferon beta or peginterferon beta-1a, or any of the other ingredients in PLEGRIDY.

Before taking PLEGRIDY, tell your healthcare provider if you:

• Are being treated for a mental illness or had treatment in the past for any mental illness, including depression and suicidal behavior
• Have or had liver problems, low blood cell counts, bleeding problems, heart problems, seizures (epilepsy), thyroid problems, or any kind of autoimmune disease
• Take prescription and over-the-counter medicines, vitamins, and herbal supplements
• Are pregnant or plan to become pregnant. It is not known if PLEGRIDY will harm your unborn baby. Tell your healthcare provider if you become pregnant during your treatment with PLEGRIDY
• Are breastfeeding or plan to breastfeed. It is not known if PLEGRIDY passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby if you use PLEGRIDY

PLEGRIDY can cause additional serious side effects including:

• Serious allergic reactions. Serious allergic reactions can happen quickly. Symptoms may include itching, swelling of the face, eyes, lips, tongue, or throat, trouble breathing, feeling faint, anxiousness, skin rash, hives, or skin bumps
• Injection site reactions. PLEGRIDY may commonly cause redness, pain or swelling at the place where the injection was given. Call your healthcare provider right away if an injection site becomes swollen and painful or the area looks infected and it does not heal within a few days. You may have a skin infection or an area of severe skin damage (necrosis) requiring treatment by a healthcare provider
• Heart problems, including congestive heart failure. While PLEGRIDY is not known to have any direct effects on the heart, some people who did not have a history of heart problems developed heart muscle problems or congestive heart failure after taking interferon beta. If you already have heart failure, PLEGRIDY may cause your heart failure to get worse. Call your healthcare provider right away if you have worsening symptoms of heart failure such as shortness of breath or swelling of your lower legs or feet while using PLEGRIDY
  • Some people using PLEGRIDY may have other heart problems, including low blood pressure, fast or abnormal heart beat, chest pain, heart attack, or a heart muscle problem (cardiomyopathy)
• Autoimmune diseases. Problems with easy bleeding or bruising (idiopathic thrombocytopenia), thyroid gland problems (hyperthyroidism and hypothyroidism), and autoimmune hepatitis have happened in some people who use interferon beta
• Blood problems and changes in your blood tests. PLEGRIDY can decrease your white blood cells or platelets, which can cause an increased risk of infection, bleeding or anemia, and can cause changes in your liver function tests. Your healthcare provider should do blood tests while you use PLEGRIDY to check for side effects
• Seizures. Some people have had seizures while taking PLEGRIDY, including people who have never had seizures before

The most common side effects of PLEGRIDY include:

• Flu-like symptoms. Many people who take PLEGRIDY have flu-like symptoms early in the course of therapy. These symptoms are not really the flu. You cannot pass it on to anyone else. Symptoms may include headache, muscle and joint aches, fever, chills or tiredness
  • You may be able to manage these flu-like symptoms by taking over-the-counter pain and fever reducers and drinking plenty of water. For many people, these symptoms lessen or go away over time

INSTRUCTIONS FOR USE

Plegridy™ (PLEGG-rih-dee)

(peginterferon beta-1a)

Injection, for Subcutaneous Use

Single-Use Prefilled Pen

Starter Pack

Read this Instructions for Use before you start using Plegridy and each time you get a refill. There may be new information. This information does not take the place of talking to your healthcare provider about your medical condition or your treatment.

Note:

• Before you use your Plegridy Pen for the first time, your healthcare provider should show you or your caregiver how to prepare and inject your Plegridy Pen the right way.
• If you experience difficulty or have questions, call 1-800-456-2255.
• Plegridy Pen is for use under the skin only (subcutaneous).
• Each Plegridy Pen can be used 1 time only. Do not share your Plegridy Pen with anyone else. By sharing the Pen, you may give an infection to them or get an infection from them.

Supplies needed for your Starter Pack for Plegridy Pen injection (See Figure A):

• 1 Starter Pack for Plegridy Pen which contains:
  • 1 Plegridy 63 mcg Pen (orange pen)
  • 1 Plegridy 94 mcg Pen (blue pen)
    

    Figure A

Additional supplies which are not included in the pack (See Figure B):

• alcohol wipe
• gauze pad
• adhesive bandage
• 1 sharps container for throwing away used needles and Plegridy Pens. See “Disposing of your used Plegridy Pens” at the end of these instructions.
• a well-lit area and a clean, flat surface to work on, like a table
  

  Figure B

Step 1: Choose your dose

• Choose the right Plegridy Pen for your dose. The Starter Pack for Plegridy Pen contains your first 2 injections to slowly adjust your dose.

When	Which Dose	Choose
Day 1 (63 mcg)	First Dose: 63 mcg	Orange Pen (See Figure C) Figure C
When	Which Dose	Choose
Day 15 (94 mcg)	Second Dose: 94 mcg	Blue Pen (See Figure D) Figure D

• Make sure you use the 63 mcg orange pen for your first dose (day 1).