FDA approved

 

Novo NordiskTRESIBA (insulin degludec injection) : Long-acting insulin analog

 RYZODEG 70/30 (insulin degludec/insulin aspart injection) : Insulin analog

Unmet Need:

  • 21 million people with diabetes in the US
  • Over time, diabetes increases the risk of heart disease, blindness, nerve and kidney damage
  • Long-acting insulins can improve blood sugar control and can reduce the risk of long-term complications

Indication: Improve glycemic control in adults with diabetes mellitus

 Efficacy : In combination with mealtime insulin

  • TRESIBA : Type 1 diabetes (3 studies, n=1,102) and Type 2 dabetes  (6 studies, n=2,702) patients , over 52 weeks
  • RYZODEG: Type 1 diabetes (1 study, n=362) and Type 2 diabetes (4 studies, n=998), over 26 weeks
  • Reductions in HbA1c (hemoglobin A1c or glycosylated hemoglobin) of  TRESIBA vs  insulin glargine met non-inferiority criteria in both populations

Safety :

  • Should not be in those with diabetic ketoacidosis
  • Hypoglycemia, allergic reactions, injection site reactions, pitting at the injection site (lipodystrophy), itching, rash, edema, and weight gain.

————————————————————

BMS

OPDIVO (nivolumab) : Programmed Death receptor (PD-1) blocking antibody

Unmet Need:

  • Melanoma is the fifth most common type of cancer in US
  • 76,100 Americans will be diagnosed with melanoma and 9,710 will die from the disease this year

Regulatory Pathway : Accelerated approval; based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials (Also breakthrough therapy designation, priority review and orphan product designation granted during development)

Indication:  In combination with ipilimumab, treatment of patients with BRAF V600 wild-type, unresectable or metastatic melanoma

Efficacy:  In combination with ipilimumab

  • Single study (n=12)  in patients with unresectable or metastatic melanoma
  • Primary endpoint : Objective Response Rate vs imipilimumab (60% vs 11%) , p<0.001
  • Progression Free Survival (27 vs 23 mo.), p<0.002
  • 14 patients : Duration of ongoing responses is at least 6 months
  • 20 patients : Duration of ongoing responses is at least 9 months

Safety:

  • Serious side effects : Severe immune-mediated side effects involving healthy organs, including the lung, colon, liver, kidneys and hormone-producing glands.
  • Common side effects : Rash, itching, cough, upper respiratory tract infections, and fluid retention (edema).

————————————————————

merck

KEYTRUDA (pembrolizumab) : Programmed Death receptor (PD-1) blocking antibody

Unmet Need:

  • Lung cancer is the leading cause of cancer death in US
  • Estimated 221,200 new diagnoses and 158,040 deaths in 2015
  • need for targeted therapy

Regulatory Pathway : Accelerated approval; based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials (Also breakthrough therapy designation, priority review and orphan product designation granted during development)

Indication :  Treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 as determined by an FDA-approved test with disease progression on or after platinum-containing chemotherapy.

Efficacy:

  • Subgroup of 61 patients enrolled within a larger multicenter, open-label, multi-part study
  • Patients had tumor proportion score (TPS) of ≥ 50% as  determined by the PD-L1 IHC 22C3 pharmDx Kit.
  • Primary endpoint : Objective Response rate (ORR) 41%; effect lasted between 2.1 and 9.1 months.

Safety:

  • Severe immune-mediated side effects occurred involving the lungs, colon and hormone-producing glands
  • Uncommon immune-mediated side effects were rash and inflammation of blood vessels (vasculitis)
  • May cause harm to a developing fetus or newborn baby
  • Guillain-Barre Syndrome also occurred.

————————————————————

FDA Voice

Why Partnerships are Key to the Science of Patient Input

FDA’s efforts to incorporate patient perspectives in drug/device assessment and regulatory process

CDRH

  • Patient Engagement Advisory Committee (PEAC) – posted previously
  •  Will provide advice on complex issues relating to medical devices, the regulation of devices, and their use by patients.

CDER, CBER

  • Patient-Focused Drug Development (PFDD) – posted previously
  • Obtain patients’ input on specific disease areas, impact on daily life, and available therapies.

Public-Private Partnerships (PPPs)

  • Medical Device Innovation Consortium (MDIC) to advance medical device regulatory science.
  • Kidney Health Initiative (KHI) to understand issues that patients with kidney diseases consider most important
  • Patient Reported Outcomes Measurement Information System (PROMIS) partnership with NIH

http://blogs.fda.gov/fdavoice/index.php/2015/10/why-partnerships-are-key-to-the-science-of-patient-input/?source=govdelivery&utm_medium=email&utm_source=govdelivery

 

 

 

 

 

 

 

 

 

Advertisements

Leave a Reply

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out /  Change )

Google photo

You are commenting using your Google account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s