Rare disease FDA decisions coming up
- Sep 30Camzyos (mavacamten) for Obstructive hypertrophic cardiomyopathy, adolescentsRead the storyDecision page
Mitapivat Faces a November 1st FDA Decision for Sickle Cell Disease on the Same Kind of Evidence Oxbryta Had
Mitapivat, a twice-daily pill already sold for 2 other blood disorders, faces an FDA decision for sickle cell disease on November 1st. Its Phase 3 trial raised hemoglobin in 40.6% of patients but did not significantly reduce pain crises, the same kind of evidence that won Oxbryta approval in 2019 before it was pulled in 2024. The trial results, the Oxbryta history, a side-by-side table of today's options, what our read of 152 sickle cell trials found about hydroxyurea, and 3 real situations.
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Atacicept vs Povetacicept for IgA Nephropathy Before the FDA Decision on November 30th
A Third of Rare Disease Trials Can Shut You Out Over a Medication You Already Take
Alpha-1 Antitrypsin Deficiency Treatment Has Meant a Weekly Infusion Since 1987, and a One-Time Gene Edit Is Now Testing That
Every FDA-approved alpha-1 antitrypsin deficiency treatment is the same idea: a weekly IV infusion of AAT protein from donated plasma. Here is how Prolastin-C, Aralast NP, Zemaira and Glassia compare, what the largest placebo-controlled trial actually showed, why none of them treats the liver, and where the pipeline stands, from a monthly lab-made AAT to a one-time base edit that lifted AAT above the historical 11 micromolar target.
Your Insurer Denied a Rare Disease Drug and Fewer Than 1 in 100 People Appeal, So Here Is the Playbook
ATTR Amyloidosis Treatment in 2026, 5 Approved Drugs Compared on Survival, Dosing and Side Effects
Before You Pick a 2027 Medicare Plan, 8 Things to Check If You Take a Rare Disease Drug
Medicare open enrollment runs October 15th to December 7th, 2026. For anyone on a rare disease drug, the 2027 Part D cap rises to $2,400, copay cards still don't apply, and infused drugs follow different rules. What to check before you switch or stay.
INO-3107 Could Become the Second Treatment for Recurrent Respiratory Papillomatosis on October 30th
What Clinical Trials Are Available for Acromegaly? 17 Are Open, and 3 Have US Sites for Patients
Lirafugratinib Is Approved as Lyrfigtu, the First FGFR2-Only Drug for Bile Duct Cancer
The FDA approved lirafugratinib as Lyrfigtu on September 23rd, 2 days early, for previously treated cholangiocarcinoma with an FGFR2 fusion. It is the third FGFR drug available for this cancer and the first built to block FGFR2 alone. What the 116-patient ReFocus cohort showed, what the label warns about, how it compares with the drugs before it, and what new research says about using it in sequence.
Camzyos Could Become the First Drug Approved for Teenagers With Obstructive HCM on September 30th
Aqneursa Is the First Approved Treatment for Ataxia-Telangiectasia, Nearly 8 Months After the Disease's Biggest Trial Failed
Emcitate Is Approved for MCT8 Deficiency, and the Trials Show It Treats the Body but Not the Brain
The FDA approved Emcitate (tiratricol) on September 28, 2026, the first treatment for MCT8 deficiency, a disease where thyroid hormone floods the body and never reaches the brain. What the US label says, what 3 trials found, what the drug does and does not fix, and how US families can get it.
Isembyld Is Approved for SMA as the First Muscle-Targeted Add-On to Spinraza and Evrysdi
UX111 Is Approved as Fayuvi, the First Treatment for Sanfilippo Syndrome, and the Label Says Exactly Who It Is For
6 Approved Drugs for IgA Nephropathy and What Their Labels Actually Promise
IgA nephropathy went from zero approved drugs to 6 in 5 years. 3 have proven they slow kidney function decline. The other 3 are approved on a urine protein number and still owe that proof. Here is the difference and why it matters to you.
Alpha-Gal Syndrome and Blood Transfusions, What Type O Patients Should Tell Their Hospital
1 Year of Palsonify and What Switching From Acromegaly Injections to a Pill Involves
We Built a Free Alpha-Gal Medication Checker
Type any drug name and see which manufacturers' versions contain gelatin, heparin, or other mammalian-derived ingredients, screened directly from FDA labels. Free, no login, and honest about what it cannot tell you.
The FDA Approved a Drug for Your Rare Disease. When Do You Actually Get It?
When a Clinical Trial Fails and It Was Your Family's Trial
The Median Rare Disease Trial Recruits at Exactly One US Site
We mapped all 20,732 US study sites behind the 6,372 recruiting trials across the 195 rare diseases Trial Friend tracks. More than half of the trials recruiting on US soil do it at a single site, 56% of recruiting trials worldwide have no US site at all, and for a patient in Wyoming the numbers get much worse.
What an FDA Clinical Hold Means When You're the One in the Trial
Zilurgisertib Could Give FOP Patients a Third Treatment on September 26th
The State of Rare Disease Clinical Trials With 75% of 2026 Behind Us
We counted every active clinical trial across the 195 rare diseases Trial Friend tracks: 9,501 studies, 5,547 recruiting, 6 diseases with nothing at all. Add 7 approvals already this year and 21 FDA decisions still to come, and 2026 is shaping up as a landmark year with an uncomfortable asterisk.
4 Rare Diseases Could Get Their First Treatment This September
Opakalim, Azetukalner, and the Race to Bring Back Epilepsy's Lost Off Switch
A New AI Tool Called MARRVEL-MCP Is Changing the Slowest Part of Rare Disease Diagnosis
A team at Baylor College of Medicine and Texas Children's Hospital just published a tool that lets a small, locally-runnable AI model interpret genetic variants for Mendelian rare diseases at 94% accuracy, up from 41% without it. The tool is open source, publicly hosted, and built on the Model Context Protocol that Anthropic released in 2024. For patients sitting on a variant of uncertain significance, this is the most important rare disease AI story of the year.
Rare Disease Diagnoses That Started With Something Else Entirely
Lone Star Tick Bites and the Rare Diseases They Cause from Alpha-Gal Syndrome to Heartland Virus
Rystiggo for Myasthenia Gravis and How It Compares to Vyvgart and Imaavy
Rystiggo (rozanolixizumab-noli) is an FcRn inhibitor approved for adults with anti-AChR or anti-MuSK antibody-positive generalized myasthenia gravis. This guide covers how it works, the MycarinG Phase 3 trial that got it approved, the side effect profile from the actual data, how it compares to Vyvgart and Imaavy, the pipeline drugs coming behind it, and how patients access it.
Alpha-Gal Syndrome: Medications, Vaccines, and Medical Products That Can Trigger a Reaction
Casgevy vs. Lyfgenia: Comparing Sickle Cell Gene Therapies
School Accommodations for a Child with a Rare Disease: A Parent's Field Guide
A practical guide for U.S. parents of children with rare diseases. Covers the differences between 504 plans, IEPs, and Individual Health Care Plans, the accommodations that actually get approved for fatigue, infection risk, mobility, cognitive load, and medical care during the day, the parent mistakes that cost families months, and the college transition where the rules quietly change.
hEDS, MCAS, POTS, MTHFR, and Tick-Borne Coinfections: When Rare Diagnoses Stack
Heparin-Induced Thrombocytopenia: A New Drug Path After 25 Years
Lonvo-Z and the First Phase 3 Win for In Vivo Gene Editing in HAE
On April 27, 2026, Intellia Therapeutics reported positive Phase 3 results for lonvo-z in hereditary angioedema. A single infusion cut HAE attacks by 87%, with 62% of treated patients going six months attack-free and therapy-free. It's the first Phase 3 win for any in vivo CRISPR therapy. For HAE patients living with chronic injections and unpredictable attacks, the implications are real.
Why Rare Disease Diagnoses Still Take 6 Years, and How Genome Sequencing Can Cut It to Weeks
The FDA's Plausible Mechanism Framework Could Unlock Gene Therapies for the Rarest Diseases
What Happens When You Take a Rare Disease to the ER
Emergency departments misdiagnose 1 in 18 patients. For rare disease patients, the odds are worse. Five conditions where the wrong ER response can turn a treatable crisis into a fatal one.
Ticks, Rare Disease, and the Coinfections Lyme Patients Have Been Told Don't Exist
How to Read a ClinicalTrials.gov Listing Without a Medical Degree
A Patient's Guide to Clinical Trial Participation
A 4-part series covering everything patients and caregivers need to consider before, during, and after expressing interest in a clinical trial. From evaluating whether a trial fits your life, to talking with your doctor, to the questions that actually matter.
Most Clinical Trial Finders Are Recruitment Tools in Disguise. We Built Something Else.
Filspari Is the First FDA-Approved Treatment for FSGS. Here's What That Actually Means for Patients.
A Quiet Crisis in Rare Disease Research: Trial Enrollment Is Slowing Just as Treatments Start Working
For decades, patients with rare diseases joined clinical trials because trials were the only option. Approved drugs have changed that math. The enrollment slowdown that has followed is reshaping how and where future therapies get developed.
Insurance Denied Your Medication. You Have More Power Than They Want You to Know.
That Drug Your Child Takes? A Voucher Worth Millions Helped Make It Happen.
Avlayah Is the First New Hunter Syndrome Treatment in 20 Years. Here's What Families Should Do Next.
Avlayah is the first Hunter syndrome therapy in 20 years that can reach the brain. For families who've been waiting, the question isn't whether this matters. The question is what to do about it.
You Got Into a Clinical Trial. Now You Have to Get There.
Dravet Syndrome Treatments in 2026: From Seizure Management to Gene Therapy
First Gene Therapy for BAG3 Heart Failure Enters Clinical Trials: What Patients Should Know
Affinia Therapeutics just received approval to begin dosing patients in the UPBEAT trial, a first-in-human gene therapy study for BAG3-associated dilated cardiomyopathy. A second company, Rocket Pharmaceuticals, is close behind. For the roughly 70,000 patients living with this genetic form of heart failure, targeted treatment has never existed until now.
Tepezza Just Got a Major Upgrade: What the New At-Home Injection Means for Thyroid Eye Disease Patients
A New ALS Trial Is Recruiting and It Learned from What Came Before
84% of Investors Are Pulling Back from Rare Disease. Here Is Why That Should Alarm Every Patient.
A coalition of nearly 100 patient groups, biotech executives, and investors sent a letter to the Trump administration urging regulatory clarity at the FDA. The numbers behind it tell a story that goes well beyond Wall Street.
From Zero Treatments to 5 in 3 Years: The IgA Nephropathy Breakthrough Nobody Saw Coming
What a $4 Million Gene Therapy Actually Costs a Family in 2026
What Happens After a Clinical Trial Ends - Your Options for Continued Access
When a clinical trial ends, the drug you have been taking may not yet be available through prescription. Several pathways exist to bridge the gap between trial completion and FDA approval - each with different eligibility, timelines, and requirements. Understanding these options matters.
A New Kind of MS Drug Just Cleared Its Biggest Hurdle: What Fenebrutinib Means for Patients
One Baby Changed Everything: How the FDA Opened the Door for Ultra-Rare Disease Treatments
What Placebos and Randomization Actually Mean for You (In Plain English)
If you have been offered a spot in a clinical trial, you have probably heard the words placebo and randomization. These concepts can sound intimidating, but they are straightforward once you understand what they actually mean for your experience as a participant.
MECP2 Gene Therapy for Rett Syndrome: What Families Should Know About the Trials in 2026
The Hidden Costs of Joining a Clinical Trial (And How to Get Help Paying for Them)
Regenxbio's Hunter Syndrome Gene Therapy Rejected: What Happened and What It Means
The FDA issued a complete response letter for RGX-121, a gene therapy designed to treat the neurological damage caused by Hunter syndrome. For the roughly 500 families in the U.S. living with MPS II, this was a major setback in a years-long wait for a treatment that could reach the brain.
When the Measuring Stick Moves: Biohaven, the FDA, and What It Means for SCA Patients
Questions about Trial Friend News and Analysis
What does Trial Friend News and Analysis cover?
Rare disease clinical trial results, FDA approval decisions, gene therapy access, drug pipelines, insurance and access problems, and treatment guides, written in plain language for patients and caregivers rather than for clinicians.
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Every post is built from primary sources: FDA announcements and drug labels, ClinicalTrials.gov records, company announcements, and peer-reviewed studies. Each post lists its sources at the end. No pharmaceutical company sponsors or influences what we publish.
Who writes Trial Friend News and Analysis?
Jason Hunter, the independent writer and editor behind Trial Friend. No drug company funds or reviews his work. He uses AI tools to help research, draft and check posts, and approves every post against its cited sources before it is published. He is not a clinician, and nothing here is medical advice.
How do you keep posts current after an FDA decision?
Posts written ahead of an FDA decision are updated in place the day the outcome is announced, and the date of the change is shown next to the publish date. The FDA calendar on Trial Friend carries each outcome with its source.
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