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Rare Disease Medications

MedRx Infusion dispenses medications for rare conditions, among them paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, neuromyelitis optica spectrum disorder and thyroid eye disease. It delivers them at no charge and, when the prescriber approves an infusion at home, arranges a licensed nurse from an accredited nursing agency.

What is a rare disease specialty pharmacy?

A rare disease specialty pharmacy dispenses medications for uncommon conditions, including some that a pharmacy may dispense only once it is certified in the medication's REMS, a safety program the FDA requires. MedRx Infusion fills rare disease medications, several of them intravenous infusions, for patients in 43 states, the District of Columbia and Puerto Rico, and therapy starts with a referral from the prescriber's office.

MedRx Infusion Clinical Pharmacy is both a specialty pharmacy and a home infusion pharmacy.

Rare disease medications MedRx dispenses, by condition

The prescriber chooses among them, and each is FDA-approved only for the conditions on its label:

  • Paroxysmal nocturnal hemoglobinuria (PNH, a blood disease in which red blood cells break down) and atypical hemolytic uremic syndrome (aHUS, small-vessel clotting that can harm the kidneys): Soliris (eculizumab) and Ultomiris (ravulizumab-cwvz), intravenous complement inhibitors that block the C5 protein.
  • Neuromyelitis optica spectrum disorder (NMOSD) with anti-aquaporin-4 antibodies: Soliris, Ultomiris and Uplizna (inebilizumab-cdon), intravenous.
  • Immunoglobulin G4-related disease: Uplizna.
  • Thyroid eye disease: Tepezza (teprotumumab-trbw), intravenous.
  • Periodic fever syndromes: Ilaris (canakinumab), subcutaneous, for cryopyrin-associated periodic syndromes, TNF receptor associated periodic syndrome, hyperimmunoglobulin D syndrome (mevalonate kinase deficiency) and familial Mediterranean fever, and for Still's disease.
  • Generalized myasthenia gravis: the FcRn blockers Vyvgart (efgartigimod alfa-fcab), Rystiggo (rozanolixizumab-noli) and Imaavy (nipocalimab-aahu), and Zilbrysq (zilucoplan), a complement inhibitor; Soliris, Ultomiris and Uplizna are also approved for it.
  • Warm autoimmune hemolytic anemia: Imaavy, for patients 12 and older who take or have taken corticosteroids.
  • Multifocal motor neuropathy: Gammagard Liquid, an intravenous immune globulin, as maintenance therapy.
  • Eosinophilic granulomatosis with polyangiitis and hypereosinophilic syndrome: Nucala (mepolizumab) and Fasenra (benralizumab), subcutaneous.
  • Autosomal dominant polycystic kidney disease at risk of rapid progression: Jynarque (tolvaptan), tablets.

Soliris, Ultomiris, Zilbrysq and Jynarque are each available only through a REMS safety program, and MedRx Infusion is certified to dispense them. The list at the foot of this page links each medication's own page.

Soliris, Tepezza and other infusions at home

When the prescriber approves home therapy for Soliris, Ultomiris, Tepezza, Vyvgart, Imaavy or Gammagard Liquid, MedRx sends a skilled, trusted and licensed nurse from an accredited nursing agency to give the infusion and watch vital signs and adverse reactions.

Uplizna's label requires close supervision by an experienced healthcare professional, premedication and at least an hour of observation after each infusion; the prescriber determines where it is given. Ilaris and Rystiggo are given by a healthcare professional, Rystiggo as a pump infusion under the skin. Zilbrysq, Nucala and Fasenra can be self-injected after training, and Jynarque tablets are taken at home, with liver blood tests the prescriber orders.

How to refer a patient with a rare disease

  1. The prescriber's office sends the prescription, recent clinical notes and laboratory results by fax to (844) 671-2601, or calls (844) 671-2600.
  2. For myasthenia gravis, NMOSD and the other nerve and muscle conditions, the office can use the Neurology referral form (PDF), which lists Soliris, Ultomiris and Vyvgart. Forms for the other specialties are on Quick Referral.
  3. MedRx's intake team checks benefits and secures any prior authorization the plan requires.
  4. The medication is delivered to the patient's door at no charge. Next-day delivery is available; timing varies by location.
  5. A MedRx pharmacist is available 24 hours a day, 7 days a week.

Insurance and cost

Prior authorization is common for rare disease medications, and MedRx's intake team handles it with the prescriber's office. MedRx also helps patients apply for copay assistance and grants, and manufacturers run support programs such as Alexion OneSource for Soliris and Ultomiris, Amgen By Your Side for Tepezza and Uplizna, My Vyvgart Path and Ilaris Companion.

Key facts

Organization
MedRx Infusion Clinical Pharmacy
What we do
A home infusion pharmacy and specialty pharmacy, with infusion nurses who visit patients at home and a pharmacist on call 24 hours a day, 7 days a week.
Where
Pharmacy at 417 N Oak St, Inglewood, CA 90302. Licensed in 43 states, the District of Columbia and Puerto Rico. See every place we serve
Accreditation
URAC (Specialty Pharmacy) and ACHC (Accreditation Commission for Health Care).
Phone, 24 hours
(844) 671-2600
Fax
(844) 671-2601

Accurate as of

Questions about rare disease medications

01What is Soliris used for?

Soliris is FDA-approved for PNH; for aHUS not caused by Shiga toxin-producing E. coli; for generalized myasthenia gravis in patients 6 and older with anti-acetylcholine receptor antibodies; and for NMOSD in adults with anti-aquaporin-4 antibodies. The prescriber decides whether it is appropriate.

02What is the difference between Soliris and Ultomiris?

Both are intravenous complement inhibitors that block C5, and both are approved for PNH, aHUS, generalized myasthenia gravis and NMOSD. Their age ranges differ, and Ultomiris is also approved, under accelerated approval, for IgA nephropathy, a kidney disease. The prescriber chooses between them.

03Can Soliris be infused at home?

It can be, when the prescriber approves home infusion. MedRx delivers Soliris at no charge and sends a nurse from an accredited nursing agency to administer it and monitor the patient during the infusion.

04What is the Soliris REMS?

The Ultomiris and Soliris REMS is the FDA-required safety program through which both medications are available. MedRx Infusion is certified to dispense Soliris and Ultomiris.

05Is there copay assistance for Soliris?

MedRx helps patients apply for copay assistance and grants, and Alexion, the manufacturer, runs a support program, Alexion OneSource. Cost depends on the patient's plan; MedRx's intake team checks coverage and obtains prior authorization before the first delivery.

Medications we fill

Each has its own page: what it is approved for, how it is given, and how to start. See every medication we fill.

Send a referral

Intake handles benefits investigation, prior authorization and scheduling.

Send a referral

Phone

(844) 671-2600

Speak with an intake coordinator

Fax

(844) 671-2601

Send a completed referral form

Forms

Referral forms

Specialty-specific PDFs