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ULTOMIRIS (ravulizumab-cwvz) injection

Alexion Pharmaceuticals
INDICATION: Treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH)
ADDRESSING UNMET NEED:
  • PNH is a rare acquired disorder that leads to the rupture or destruction of red blood cells (hemolysis)
  • Prior to this approval, treatment required every two weeks, which can be burdensome for patients and families
  • Novel formulation so patients only need treatment every eight weeks, without compromising efficacy

MECHANISM OF ACTION: Terminal complement inhibitor specifically binds to complement protein C5 with high affinity; inhibits terminal complement-mediated intravascular hemolysis in patients with PNH

EFFICACY & SAFETY:
  • First randomized clinical trial, n=246 PNH patients (treatment naïve), Ultomiris vs. eculizumab, current standard of care for PNH
  • Non-inferiority on endpoints: Transfusion avoidance and hemolysis directly measured by normalization of LDH levels
  • Second randomized trial, n=195 PNH patients clinically stable after eculizumab treatment, Ultomiris vs. continue eculizumab
  • Similarity on several clinical measures including hemolysis and avoiding transfusion
  • Common side effects: Headache, upper respiratory infection
  • Boxed Warning: Risk of life-threatening meningococcal infections and sepsis
REGULATORY PATHWAY: BLA
  • Priority Review designation, Orphan Drug designation
  • Postmarketing commitments: Data from clinical trials in patients with paroxysmal nocturnal hemoglobinuria (ALXN1210-PNH-301 and ALXN1210-PNH-302) to include the extension period of up to 2 years of follow-up

Personal Genome Service (PGS) Genetic Health Risk Report for
MUTYH-Associated Polyposis for Cervical Cancer screening

23andMe

INTENDED USE: PGS uses qualitative genotyping to detect select clinically relevant variants in genomic DNA isolated from human saliva collected from individuals ≥18 years for the purpose of reporting and interpreting genetic health risks, including the 23andMe PGS Genetic Health Risk Report for MUTYH-Associated Polyposis (MAP)

INDICATION FOR USE: The 23andMe PGS Genetic Health Risk Report for MUTYHAssociated Polyposis is indicated for reporting of the Y179C and the G396D variants in the MUTYH gene. The report describes if a person is at increased risk of developing colorectal cancer. The two variants included in this report are most common and best studied in people of Northern European descent and may not represent the majority of the MUTYH variants found in people of other ethnicities. The test report does not describe a person’s overall risk of developing any type of cancer, and the absence of a variant tested does not rule out the presence of other variants that may be cancer-related.

This test is not a substitute for visits to a healthcare provider for recommended screenings or appropriate follow-up and should not be used to determine any treatments.

DESCRIPTION:

  • The core components of the PGS are unchanged from the de novo authorization for the predicate device, the PGS Genetic Health Risk Report for BRCA1/BRCA2 and also the additional de novo Authorizations of the PGS for Carrier Screening Tests for Bloom Syndrome and PGS Genetic Health Risk Reports including saliva collection kit, reagents and beadchip, instrumentation, software, test processes, procedures
  • Novel components: (a) specific variants to be reported (b) qualitative reporting of risk for MAP, as opposed to the BRCA related cancer risks

REGULATORY PATHWAY: Traditional 510(k)

  • Trade/Device Name: MUTYH-Associated Polyposis (MAP)
  • Regulation Number: 21 CFR 866.6090
  • Regulation Name: Cancer Predisposition Risk Assessment System
  • Regulatory Class: Class II
  • Product Code: QAZ

SUMMARY


Image credits: Alexion, 23andMe

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