Specialty Pipeline Quarterly Update: June 2022

This pipeline wrap-up provides a review of newly approved specialty drugs, recent specialty drug launches, new indications and news of note on specialty drugs in the approval process. See separate articles for pipeline information on traditional drugs.

New Drug Information

• Fylnetra^® (pegfilgrastim-pbbk): The FDA approved Amneal’s Fylnetra (pegfilgrastim-pbbk) a leukocyte growth factor biosimilar to Neulasta^® (pegfilgrastim) used to reduce the incidence of neutropenia in patients undergoing chemotherapy. Fylnetra is the fifth Neulasta biosimilar, other biosimilars include: Mylan/Biocon’s Fulphila^® (pegfilgrastim-jmdb), Pfizer’s Nyvepria™ (pegfilgrastim-apgf), Coherus BioSciences’ Udenyca^® (pegfilgrastim-cbqv) and Sandoz’s Ziextenzo^® (pegfilgrastim-bmez) which are already available in the U.S. None of the biosimilars are interchangeable with Neulasta or with each other.¹ According to IQVIA^®, U.S. annual sales for pegfilgrastim for the 12 months ended March 2022 were $3.1 billion, $1.0 billion of which represented biosimilar sales.² Amneal is planning on launching Fylnetra the second half of 2022 with pricing to follow.
• Amvuttra^® (vutrisiran): The FDA approved Amvuttra (vutrisiran), an RNAi therapeutic, for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. hATTR amyloidosis is a rare, inherited, rapidly progressive, and fatal disease with debilitating polyneuropathy manifestations, for which there are few treatment options. Amvuttra is administered via subcutaneous healthcare administered injection, once every three months. The FDA approval is based on positive 9-month results from the HELIOS-A Phase 3 study, where Amvuttra significantly improved the signs and symptoms of polyneuropathy. Amvuttra met the primary endpoint of the study, the change from baseline in the modified Neuropathy Impairment Score + 7 (mNIS+7) at 9 months. Treatment with Amvuttra resulted in a 2.2 point mean decrease (improvement) in mNIS+7 from baseline as compared to a 14.8 point mean increase (worsening) reported for the external placebo group, resulting in a 17.0 point mean difference relative to placebo; by 9 months, 50 percent of patients treated with Amvuttra experienced improvement in neuropathy impairment relative to baseline.³ Amvuttra is scheduled to launch early July with pricing to follow.
• Tyvaso DPI^® (treprostinil dry powder for inhalation): The FDA approved a new formulation of United Therapeutics’ Tyvaso DPI (treprostinil dry powder for inhalation) for treatment of pulmonary arterial hypertension (PAH; WHO Group 1 pulmonary hypertension) and pulmonary hypertension associated with interstitial lung disease (PH-ILD; WHO Group 3 pulmonary hypertension). Tyvaso DPI was approved based Phase 1 BREEZE study that demonstrated that the transition from Tyvaso inhalation solution to Tyvaso DPI was safe and well tolerated.⁴ The Tyvaso DPI Inhaler can be used for up to seven days from the date of first use, after which it must be discarded and replaced with a new inhaler. Tyvaso DPI is expected to be available in June 2022 with pricing to follow.

New Indications 

• Evrysdi^® (risdiplam): The FDA expanded Genentech’s Evrysdi (risdiplam) indication to include pre-symptomatic babies under 2 months of age with spinal muscular atrophy (SMA).
• Tibsovo^® (ivosidenib): The FDA expanded the indication for Agios Pharmaceuticals’ Tibsovo (ivosidenib) to be used in combination with azacitidine for newly diagnosed acute myeloid leukemia (AML) with a susceptible IDH1 mutation, as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.
• Vidaza^® (azacitadine): Bristol Myers Squibb’s Vidaza (azacitadine) has been approved by the FDA to include a new indication for treatment of pediatric patients aged one month and older with newly diagnosed juvenile myelomonocytic leukemia (JMML).
• Dupixent^® (dupilumab): The FDA approved an additional indication for Sanofi Pharmaceuticals’ Dupixent (dupilumab) to treat eosinophilic esophagitis (EoE) in adults and pediatric patients 12 years and older weighing at least 40 kilograms (which is about 88 pounds).
• Olumiant^® (baricitinib): The FDA expanded the indication of Lilly and Incyte’s Olumiant (baricitinib) to include the treatment of COVID-19 in hospitalized adults requiring supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO). Additionally, the FDA added the treatment of alopecia areata to the indications for Olumiant.

June News

• “Two years after the FDA turned back BioMarin’s push for a landmark accelerated approval of their hemophilia A gene therapy valrox, regulators are asking for still more info on the therapy — and once again delaying any prospective launch, this time into next year. Buried under the latest set of extended data from their Phase 1/2 trial of valrox (valoctocogene roxaparvovec), which continues to demonstrate its effectiveness in preventing bleeds up to six years after initial treatment, the biotech spelled out the reasons why they’re shoving back an expected resubmission from June to September. The agency wants more data and analysis, says the biotech, but isn’t asking for any new studies. And the news will likely enhance some deep-seated concerns that the FDA appears to be in no rush to approve any gene therapy overshadowed by questions of durability and safety.”⁵
• “The four companies working on gene therapies for Duchenne muscular dystrophy (DMD) have had another thing in common besides the modality and disease they’re working on: serious adverse events. Rather than continue to plug away at individual studies and try to figure out why these side effects were happening in certain patients, Pfizer, Sarepta, Genethon and Solid Biosciences teamed up for a pooled safety analysis (PDF) that was presented this week at the American Society of Gene and Cell Therapy meeting. The companies shared clinical and laboratory data and put together a panel of experts to take a look with the goal of minimizing further medical complications.”⁶
• “Due to safety concerns, the U.S. Food and Drug Administration (FDA) has withdrawn its approval for the cancer medicine Ukoniq (umbralisib). Ukoniq was approved to treat two specific types of lymphoma: marginal zone lymphoma (MZL) and follicular lymphoma (FL). Updated findings from the UNITY-CLL clinical trial continued to show a possible increased risk of death in patients receiving Ukoniq. As a result, we determined the risks of treatment with Ukoniq outweigh its benefits.”⁷
• “Roughly 24 hours after Canadian regulators gave Amylyx Pharmaceuticals the thumbs-up for its new ALS drug, ICER is out with a new analysis trying to determine whether the drug would be cost effective. And the short answer is no — for now. Though Amylyx has not disclosed how much it intends to charge for the drug, branded as Albrioza, ICER pegged its estimated cost to an older ALS medicine, edaravone. If Albrioza costs $169,000 per year, as edaravone currently does, the drug would not be cost effective to the US healthcare system, and far from it, ICER says. Using a measurement known as quality-adjusted life year, or QALY, ICER said in its draft report that Albrioza would only be cost effective if priced at $13,700 annually — less than 10% of edaravone’s price point. QALY represents the best estimation of the value of a year of good health, Rind said, combining length of life and quality of life into a single measure when given a certain treatment. In general, ICER uses $150,000 as the benchmark for how much US patients or insurers are willing to pay for an extra year of good health, Rind said. Albrioza would be more cost effective, however, than the oral formulation of edaravone, recently approved by the FDA, ICER says oral edaravone should be priced at $2,200 per year to achieve cost effectiveness.”⁸