Rare Diseases/Orphan Drugs

New FDA Approvals: FDA Expands Patient Population for Diacomit

July 14: The FDA expanded the patient population of Biocodex, Inc.’s Diacomit (stiripentol) for the treatment of seizures associated with Dravet syndrome in people between the ages of 6 months to 2 years and weighing at least 7 kg who are taking clobazam. The agency first approved the treatment on Aug. 20, 2018. The drug is available as a capsule and an oral suspension. Dosing is 50 mg/kg/day for both routes of administration. Drugs.com lists the price of 60 250 mg capsules and 60 250 mg powder for reconstitution as more than $1,589.

July 14: The FDA expanded the label of Pfizer Inc.’s Xalkori (crizotinib) to include the treatment of people at least 1 year old with unresectable, recurrent or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors. The agency initially approved the kinase inhibitor on Aug. 26, 2011. The FDA gave the agent orphan drug designation and granted the application priority review; that review used the Assessment Aid, a voluntary submission from the applicant to assist the FDA in its analysis. Dosing for the newest indication of the capsule in adults is 250 mg twice daily. The recommended pediatric dosage is 280 mg.m2 twice daily based on body surface area. Drugs.com lists the price of 60 250 mg capsules as more than $20,657.

Researchers Examine CF, UC/Crohn’s Adherence, Say Specialty Pharmacies ‘Could Help Reduce Medical Burden’

Two recent studies of specialty-drug treated conditions examined the impact of adherence on hospitalizations and medical costs. Findings of the studies — one on cystic fibrosis (CF) and the other on ulcerative colitis (UC)/Crohn’s disease — from AllianceRx Walgreens Prime (which changed its name to AllianceRx Walgreens Pharmacy in late June) demonstrate the importance of specialty pharmacy interventions in helping keep patients adherent to therapy.

The study posters were presented at the recent International Society for Pharmacoeconomics and Outcomes Research 2022 Conference held in Washington, D.C.

Expanded Label Puts SMA Drugs on Even Terms for Youngest Patients

A recent FDA approval of a label expansion put the three marketed therapies for spinal muscular atrophy (SMA) on equal footing for the youngest patients. A recent survey found that many payers are covering sequential use of the costly agents, including a gene therapy.

The FDA initially approved Evrysdi (risdiplam) from Roche Group member Genentech USA, Inc. on Aug. 7, 2020, for the treatment of SMA in people at least 2 months old. On May 30, 2022, the agency expanded the drug’s label to include the treatment of babies less than 2 months old. The medication is an oral solution administered by mouth or feeding tube and can be administered by a patient or caregiver at home after a recommended consultation with a health care professional prior to the first dose.

New FDA Approvals: FDA Grants Additional Indication to CellCept

June 6: The FDA expanded the label of Roche Group member Genentech USA, Inc.’s CellCept (mycophenolate mofetil) to include, in combination with other immunosuppressants, prophylaxis of organ rejection in people at least 3 months old who have received an allogenic heart transplant or an allogenic liver transplant. The agency first approved the drug on May 3, 1995. Dosing for the newest uses is based on body surface area and indication. The drug is available as a capsule, tablet, oral suspension and intravenous injectable. Website GoodRx.com lists the price of 60 500 mg tablets as more than $1,070.

June 7: The FDA granted another indication to Sanofi and Regeneron Pharmaceuticals, Inc.’s Dupixent (dupilumab) for the treatment of moderate-to-severe atopic dermatitis in people between the ages of 6 months and 5 years whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. The agency initially approved the subcutaneous injectable on March 28, 2017. The FDA gave the new indication priority review. Dosing for people weighing 5 kg to less than 15 kg is 200 mg every four weeks; for those weighing 15 kg to less than 30 kg, dosing is 300 mg every four weeks. The drug’s list price, regardless of dose, is $3,384.83 per carton, which includes either two prefilled pens or two prefilled syringes.

Optum Looks to Create Industry Standard for Genetic Testing Management

Optum says it hopes to create the industry standard for genetic testing benefit management programs — across all business lines and payers nationwide — with a solution that the UnitedHealth Group subsidiary is launching with Avalon Healthcare Solutions using genetic testing codes and other assets licensed from Palmetto GBA.

The solution is part of a comprehensive laboratory benefit management program that the companies unveiled June 22. Although 99% of all lab tests conducted today are routine tests, 10 new genetic tests are coming to the market each day, says John Hoffman, vice president of payer market at Optum. He and colleagues from Optum and Avalon outlined the solution at AHIP 2022 in Las Vegas.


People With Rare Diseases Face Challenges, Require Support

In the U.S., orphan diseases are conditions impacting fewer than 200,000 people. There are more than 7,000 of these rare conditions affecting an estimated 30 million Americans — and more than 300 million people globally — and new diseases continue to be discovered. Most of them are inherited conditions caused by gene mutations, but some can be caused by environmental factors. These diseases may be serious and even life-threatening, and about half of them affect children.

Before the Orphan Drug Act was passed in 1983, not much research was done into treatments for rare diseases. But that law created financial incentives for pharmaceutical manufacturers, and since then, hundreds of orphan drugs have been developed. As of early 2020, the FDA had approved therapies for more than 800 rare diseases.

New FDA Approvals: FDA Grants Additional Indication to Olumiant

May 10: The FDA granted full approval to Eli Lilly and Co.’s Olumiant (baricitinib) for the treatment of COVID-19 in hospitalized adults requiring supplemental oxygen, noninvasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation. The agency first approved the Janus kinase (JAK) inhibitor on May 31, 2018. The drug has been available for COVID treatment since Nov. 19, 2020, under Emergency Use Authorization (EUA). The EUA remains in place for hospitalized people between the ages of 2 and 18 years old who require various degrees of oxygen support. The recommended dose of the tablet for the newest use is 4 mg once daily for 14 days or until hospital discharge, whichever occurs first. Alternative modes of administration via oral dispersion, gastrostomy tube, nasogastric tube or orogastric tube are available. The drug’s list price for a 30-day supply of 2 mg tablets is $2,497.20.

New FDA Approvals: The FDA Approved Amneal’s Alymsys

April 13: The FDA approved Amneal Pharmaceuticals, Inc.’s Alymsys (bevacizumab-maly) for the treatment of multiple conditions: (1) first- or second-line treatment of metastatic colorectal cancer in combination with intravenous fluorouracil-based chemotherapy; (2) second-line treatment of metastatic colorectal cancer in combination with fluoropyrimidine-irinotecan or fluoropyrimidine-oxaliplatin chemotherapy in people who have progressed on a first-line bevacizumab product; (3) first-line treatment of unresectable, locally advanced, recurrent or metastatic non-squamous non-small cell lung cancer in combination with carboplatin and paclitaxel; (4) recurrent glioblastoma in adults; (5) metastatic renal cell carcinoma in combination with interferon alfa; (6) persistent, recurrent or metastatic cervical cancer in combination with paclitaxel and cisplatin or paclitaxel and topotecan; and (7) epithelial ovarian, fallopian tube or primary peritoneal cancer in combination with paclitaxel, pegylated liposomal doxorubicin or topotecan for platinum-resistant recurrent disease in people receiving no more than two prior chemotherapy regimens. The vascular endothelial growth factor inhibitor is the third biosimilar of Roche Group member Genentech USA, Inc.’s Avastin (bevacizumab) that the agency has approved. Dosing of the intravenous infusion is based on indication.

Centene Plans to Sell Magellan Rx, PANTHERx Rare for $2.8 Billion

Centene Corp. has agreed to sell two of its pharmacy businesses, Magellan Rx and PANTHERx Rare, in separate transactions as part of the insurer’s decision last year to exit the PBM industry.

Prime Therapeutics, a PBM jointly owned by 19 Blue Cross and Blue Shield affiliates, is acquiring Magellan Rx for about $1.35 billion in a deal that’s expected to close in the fourth quarter, while a joint venture of the Vistria Group, General Atlantic and Nautic Partners is buying PANTHERx Rare for $1.45 billion in a deal that’s expected to be completed in the next two to four months.

CMS Rule on Pharma Patient-Assistance Programs Could Cut Back on Aid

CMS’s stance has long been that manufacturer-provided assistance given to patients is excluded from best price and average manufacturer price (AMP) calculation for prescription drugs. However, the rise of copayment accumulators and maximizers — and health insurers’ subsequent taking of this assistance rather than allowing it to count toward patients’ deductibles and out-of-pocket maximums — have caused the agency to rethink its position. A rule slated to take effect at the beginning of 2023 would reverse that longtime approach, potentially resulting in increased patient out-of-pocket costs for drugs and pharma companies being on the hook for ensuring they know exactly where their assistance is going, industry experts tell AIS Health, a division of MMIT.

The Medicaid rebate rule allows state Medicaid programs to get the same discounts on drug prices that manufacturers offer commercial plans purchasing prescription drugs. Manufacturers pay rebates to Medicaid programs that are calculated based on drugmakers’ best price, which is the lowest price the manufacturer gives to most providers of health care services or items, including hospitals, HMOs and MCOs — but not patients. It includes any price adjustments, such as discounts and rebates, but not manufacturer-provided assistance to patients.